Scholars@Duke
Kris Mahadeo

Kris Mahadeo

Professor of Pediatrics
Pediatrics, Transplant and Cellular Therapy
kris.mahadeo@duke.edu
Pediatric Transplant and Cellular Therapy, 2400 Pratt Street, Durham, NC 27705

Selected Publications

Erratum to

Journal Article Transplant Cell Ther · October 30, 2025 Full text Link to item Cite

Bezlotoxumab as Treatment for Recurrent/Chronic Clostridioides difficile Infection in Pediatric Stem Cell Transplant Recipients: A Multi-Institutional Experience.

Journal Article Pediatr Blood Cancer · October 2025 Clostridioides difficile infection (CDI) represents a significant health risk to pediatric stem cell transplant (SCT) patients. In these patients, recurrent CDI is common, requiring prolonged antibiotic usage and increased hospitalization. Bezlotoxumab, a ... Full text Link to item Cite

Integrating pulmonary and systemic transcriptomes to characterize lung injury after pediatric hematopoietic stem cell transplant.

Journal Article JCI Insight · September 9, 2025 Hematopoietic stem cell transplantation (HCT) is a potentially life-saving therapy but can lead to lung injury due to chemoradiation toxicity, infection, and immune dysregulationWe previously showed that bronchoalveolar lavage (BAL) transcriptomes represen ... Full text Link to item Cite

International expert consensus statement on PICU admission and early critical care management for paediatric patients following haematopoietic cell transplant and immune effector cell therapy.

Journal Article Lancet Child Adolesc Health · June 2025 Advances in paediatric haematopoietic cell transplantation strategies using immune-effector cells (HCT-IEC) and in intensive care management have improved survival expectations for patients with malignant and non-malignant diseases. However, critical illne ... Full text Link to item Cite

Haploidentical Bone Marrow Transplantation for Sickle Cell Disease.

Journal Article NEJM Evid · March 2025 BACKGROUND: Related human leukocyte antigen (HLA)-haploidentical bone marrow transplantation (BMT) with posttransplant cyclophosphamide may be curative for sickle cell disease. However, graft failure, severe graft-versus-host disease (GVHD), infections, an ... Full text Link to item Cite

Newborn Screening for Hurler Syndrome Facilitates Early Transplant and Good Outcomes.

Journal Article Pediatr Neurol · February 2025 BACKGROUND: Hematopoietic cell transplantation (HCT) is the standard of care treatment for children with Hurler syndrome (HS). This study describes the impact of newborn screening (NBS) on HCT outcomes for these patients. METHODS: Retrospective study of HS ... Full text Link to item Cite

Safety and feasibility of umbilical cord blood transplantation in children with neuronal ceroid lipofuscinosis: a retrospective study.

Journal Article Stem Cells Transl Med · January 17, 2025 Ceroid lipofuscinosis neuronal (CLN) encompasses rare inherited neurodegenerative disorders that present in childhood with clinical features including epilepsy, psychomotor delay, progressive vision loss, and premature death. Published experience utilizing ... Full text Link to item Cite

A reduced-toxicity myeloablative conditioning approach for hematopoietic cell transplant in juvenile myelomonocytic leukemia.

Journal Article Front Oncol · 2025 INTRODUCTION: Allogeneic hematopoietic cell transplantation (HCT) is a potentially curative treatment for most children with juvenile myelomonocytic leukemia (JMML), but overall survival remains poor at 50%. Given its rarity and heterogeneity, there is no ... Full text Link to item Cite

Clinician Adherence to Screening and Preventive Practices for Non-Malignant Late Effects Among Allogeneic Hematopoietic Cell Transplant Survivors in the United States

Conference Blood · November 5, 2024 Background: Survivors of allogeneic hematopoietic cell transplantation (HCT) are at risk for treatment-related late effects. Published recommendations for screening/prevention of late com ... Full text Cite

Real-World Clinician Adherence to Screening and Preventive Practices Guidelines for Subsequent Neoplasms Among Allogeneic Hematopoietic Cell Transplant Survivors in the United States

Conference Blood · November 5, 2024 Background: Prior studies have reported variable adherence to screening/prevention guidelines from allogeneic hematopoietic cell transplant (HCT) survivors' perspective (Khera N et al. Bi ... Full text Cite

Critical Care Support in Infants Post-Allogeneic HCT: Large Single-Center Cohort Experience

Conference Blood · November 5, 2024 IntroductionAllogeneic hematopoietic cell transplantation (HCT) is a potentially life-saving treatment for various diseases observed in childhood but m ... Full text Cite

Cultured Thymus Tissue Implantation for Congenital Athymia Post FDA Approval: Auto-Immunity and Novel Genetic Mutations

Conference Blood · November 5, 2024 IntroductionCongenital athymiais an ultra-rare cellular immune deficiency characterized by absent T cell thymic emigrants, significant T cell dysfuncti ... Full text Cite

Updated Clinical Results: A Multicenter, Open-Label, Phase 3 Study of Tabelecleucel for Solid Organ or Allogeneic Hematopoietic Cell Transplant Recipients with Epstein-Barr Virus-Driven Post Transplant Lymphoproliferative Disease after Failure of Rituximab or Rituximab Plus Chemotherapy

Conference Blood · November 5, 2024 Background: The ALLELE trial (NCT03394365) is investigating the effects of tabelecleucel, an off-the-shelf allogeneic Epstein-Barr virus (EBV)-specific cytotoxic T cell therapy, to treat ... Full text Cite

Menin inhibitors in pediatric acute leukemia: a comprehensive review and recommendations to accelerate progress in collaboration with adult leukemia and the international community.

Journal Article Leukemia · October 2024 Aberrant expression of HOX and MEIS1 family genes, as seen in KMT2A-rearranged, NUP98-rearranged, or NPM1-mutated leukemias leads to arrested differentiation and leukemia development. HOX family genes are essential gatekeepers of physiologic hematopoiesis, ... Full text Link to item Cite

Humoral Immunity and Antibody Responses against Diphtheria, Tetanus, and Pneumococcus after Immune Effector Cell Therapies: A Prospective Study.

Journal Article Vaccines (Basel) · September 19, 2024 Patients undergoing immune effector cell therapy (IECT) are at high risk for infections. We assessed seropositivity against pneumococcus, tetanus, and diphtheria in patients before and after IECT and the patients' response to vaccination. We enrolled patie ... Full text Link to item Cite

Pathobiological signatures of dysbiotic lung injury in pediatric patients undergoing stem cell transplantation.

Journal Article Nat Med · July 2024 Hematopoietic cell transplantation (HCT) uses cytotoxic chemotherapy and/or radiation followed by intravenous infusion of stem cells to cure malignancies, bone marrow failure and inborn errors of immunity, hemoglobin and metabolism. Lung injury is a known ... Full text Link to item Cite

Tabelecleucel for EBV+ PTLD after allogeneic HCT or SOT in a multicenter expanded access protocol.

Journal Article Blood Adv · June 25, 2024 Patients with Epstein-Barr virus (EBV)-positive posttransplant lymphoproliferative disease (EBV+ PTLD) in whom initial treatment fails have few options and historically low median overall survival (OS) of 0.7 months after allogeneic hematopoietic cell tran ... Full text Link to item Cite

MDB-83. OUTCOMES OF INFANTS AND YOUNG CHILDREN WITH NEWLY DIAGNOSED SHH MEDULLOBLASTOMA TREATED ON THE NEXT CONSORTIUM “HEAD START” 4 PROTOCOL

Conference Neuro-Oncology · June 18, 2024 AbstractBACKGROUNDInfants and young children with SHH medulloblastoma were demonstrated to have a favorable outcome on “Head Start (HS ... Full text Cite

Retrospective analysis of veno-occlusive disease/sinusoidal obstruction syndrome in paediatric patients undergoing hematopoietic cell transplantation -a multicentre study.

Journal Article Lancet Reg Health Am · May 2024 BACKGROUND: Sinusoidal obstruction syndrome is a potentially fatal complication following hematopoietic cell transplantation, high-intensity chemotherapies and increasingly seen with calicheamicin based leukemia therapies. Paediatric specific European Soci ... Full text Link to item Cite

Tabelecleucel for allogeneic haematopoietic stem-cell or solid organ transplant recipients with Epstein-Barr virus-positive post-transplant lymphoproliferative disease after failure of rituximab or rituximab and chemotherapy (ALLELE): a phase 3, multicentre, open-label trial.

Journal Article Lancet Oncol · March 2024 BACKGROUND: Survival in Epstein-Barr virus (EBV)-positive post-transplant lymphoproliferative disease following haematopoietic stem-cell transplant (HSCT) or solid organ transplant (SOT) is poor after failure of initial therapy, indicating an urgent need f ... Full text Link to item Cite

Cell membrane-anchored and tumor-targeted IL-12 T-cell therapy destroys cancer-associated fibroblasts and disrupts extracellular matrix in heterogenous osteosarcoma xenograft models.

Journal Article J Immunother Cancer · January 9, 2024 BACKGROUND: The extracellular matrix (ECM) and cancer-associated fibroblasts (CAFs) play major roles in tumor progression, metastasis, and the poor response of many solid tumors to immunotherapy. CAF-targeted chimeric antigen receptor-T cell therapy cannot ... Full text Link to item Cite

Characteristics and outcomes of children, adolescents and young adults with relapsed/refractory non-hodgkin lymphoma undergoing autologous stem cell transplant.

Journal Article BMC Cancer · December 20, 2023 BACKGROUND: There is paucity of data regarding outcomes of children, adolescents and young adults (CAYA) patients with non-Hodgkin lymphoma (NHL) undergoing autologous stem cell transplantation (ASCT). METHODS: Patients aged 0-39 years undergoing first ASC ... Full text Link to item Cite

Reduced Intensity Haploidentical Bone Marrow Transplantation in Adults with Severe Sickle Cell Disease: BMT CTN 1507

Conference Blood · November 21, 2023 Background: Allogeneic hematopoietic stem-cell transplantation has curative potential for sickle cell disease (SCD). Event-free survival (EFS) in children with SCD is >90% after a bone marrow transplant (BMT) fr ... Full text Cite

The Impact of Leukapheresis Collection on Patients Undergoing Chimeric Antigen T-Cell Receptor Therapy with Tisagenlecleucel

Conference Blood · November 2, 2023 IntroductionAutologous chimeric antigen receptor T-cell therapy (CAR-T) has revolutionized the treatment of acute lymphoblastic leukemia (ALL) in children and young adolescents, with an ... Full text Cite

Blastic plasmacytoid dendritic cell neoplasm: a comprehensive review in pediatrics, adolescents, and young adults (AYA) and an update of novel therapies.

Journal Article Leukemia · September 2023 Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematologic malignancy that can involve the bone marrow, peripheral blood, skin, lymph nodes, and the central nervous system. Though more common in older adults, BPDCN has been reported across ... Full text Link to item Cite

Immune Effector Cell-Associated Hemophagocytic Lymphohistiocytosis-Like Syndrome.

Journal Article Transplant Cell Ther · July 2023 T cell-mediated hyperinflammatory responses, such as cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), are now well-established toxicities of chimeric antigen receptor (CAR) T cell therapy. As the field of ... Full text Link to item Cite

A phase 1 dose escalation trial of ex-vivo expanded allogeneic cord blood–derived natural killer cell immunotherapy for pediatric solid tumor malignancies.

Conference Journal of Clinical Oncology · June 1, 2023 2546 Background: Despite aggressive multimodality therapy, survival outcomes of most patients with recurrent/refractory solid tumors remain dismal. Preclinical data have shown that natural killer (NK) cells play a significant rol ... Full text Cite

Outcomes following posttransplant virus-specific T-cell therapy in patients with sickle cell disease.

Journal Article Blood Adv · May 23, 2023 Hematopoietic stem cell transplantation (HSCT) is being increasingly used as a curative approach for sickle cell disease (SCD). With the risk of graft-versus-host disease (GVHD), especially in the human leukocyte antigen-mismatched donors, intense immunosu ... Full text Link to item Cite

Extracorporeal membrane oxygenation in adults receiving haematopoietic cell transplantation: an international expert statement.

Journal Article Lancet Respir Med · May 2023 Combined advances in haematopoietic cell transplantation (HCT) and intensive care management have improved the survival of patients with haematological malignancies admitted to the intensive care unit. In cases of refractory respiratory failure or refracto ... Full text Link to item Cite

Characteristics and Outcomes of Children, Adolescents and Young Adults with Relapsed/Refractory Non-Hodgkin Lymphoma Undergoing Autologous Stem Cell Transplant.

Journal Article Res Sq · February 7, 2023 There is paucity of data regarding outcomes of children, adolescents and young adults (CAYA) patients with non-Hodgkin lymphoma (NHL) undergoing autologous hematopoietic stem cell transplantation (ASCT). We analyzed 222 patients aged 0-39 years undergoing ... Full text Link to item Cite

Prior COVID-19 infection may increase risk for developing endothelial dysfunction following hematopoietic cell transplantation

Journal Article Frontiers in Oncology · January 17, 2023 Endothelial dysfunction underlies many of the major complications following hematopoietic cell transplantation (HCT), including transplant-associated thrombotic microangiopathy (TA-TMA), veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS), and ... Full text Cite

Miscellaneous Complications of Hematopoietic Cellular Transplantation

Chapter · January 1, 2023 Advancements in hematopoietic cell transplantation (HCT) have been associated with expanding indications and eligibility, alternative donor options, and improvement in overall survival. Yet, HCT for hematologic malignancies are still associated with the po ... Full text Cite

A multicenter study of ICU resource utilization in pediatric, adolescent and young adult patients post CAR-T therapy

Journal Article Frontiers in Oncology · October 24, 2022 Tisagenlecleucel is associated with remarkable outcomes in treating patients up to the age of 25 years with refractory B-cell acute lymphoblastic leukemia (ALL). Yet, due to unique and potentially life-threatening complications, access remains limited to h ... Full text Cite

The Evolution of Chimeric Antigen Receptor T-Cell Therapy in Children, Adolescents and Young Adults with Acute Lymphoblastic Leukemia

Journal Article Biomedicines · September 1, 2022 Chimeric antigen receptor T-cell (CAR T) therapy is a revolutionary treatment for pediatric, adolescent and young adult patients (AYA) with relapsed/refractory B-cell acute lymphoblastic leukemia. While the landscape of immunotherapy continues to rapidly e ... Full text Cite

Noninvasive Ventilation Exposure Prior to Intubation in Pediatric Hematopoietic Cell Transplant Recipients.

Journal Article Respir Care · September 2022 BACKGROUND: Noninvasive ventilation (NIV) has become more studied in immunocompromised patients. However, it has not been studied in hematopoietic cell transplantation (HCT) recipients, who have higher mortality and higher pulmonary complication rates than ... Full text Link to item Cite

Post-Transplant Cyclophosphamide after Matched Sibling and Unrelated Donor Hematopoietic Stem Cell Transplantation in Pediatric Patients with Acute Myeloid Leukemia.

Journal Article Int J Mol Sci · August 6, 2022 Non-relapse mortality due to GVHD and infections represents a major source of morbidity and mortality in pediatric HSCT recipients. Post-transplant cyclophosphamide (PTCy) has emerged as an effective and safe GVHD prophylaxis strategy, with improved GVHD a ... Full text Link to item Cite

Management of Aggressive Non-Hodgkin Lymphomas in the Pediatric, Adolescent, and Young Adult Population: An Adult vs. Pediatric Perspective

Journal Article Cancers · June 1, 2022 Non-Hodgkin lymphoma (NHL) is a broad entity which comprises a number of different types of lymphomatous malignancies. In the pediatric and adolescent population, the type and prognosis of NHL varies by age and gender. In comparison to adults, pediatric an ... Full text Cite

High-Risk Acute Myeloid Leukemia: A Pediatric Prospective

Journal Article Biomedicines · June 1, 2022 Pediatric acute myeloid leukemia is a clonal disorder characterized by malignant trans-formation of the hematopoietic stem cell. The incidence and the outcome remain inferior when compared to pediatric ALL, although prognosis has improved in the last decad ... Full text Cite

Improved outcomes of high-risk relapsed Hodgkin lymphoma patients after high-dose chemotherapy: a 15-year analysis.

Journal Article Haematologica · April 1, 2022 High-dose chemotherapy and autologous stem-cell transplant (HDC/ASCT) is standard treatment for chemosensitive relapsed classical Hodgkin lymphoma, although outcomes of high-risk relapse (HRR) patients remain suboptimal. We retrospectively analyzed all HRR ... Full text Link to item Cite

Candidacy for Extracorporeal Life Support in Children After Hematopoietic Cell Transplantation: A Position Paper From the Pediatric Acute Lung Injury and Sepsis Investigators Network's Hematopoietic Cell Transplant and Cancer Immunotherapy Subgroup.

Journal Article Pediatr Crit Care Med · March 1, 2022 OBJECTIVES: The last decade has seen improved outcomes for children requiring extracorporeal life support as well as for children undergoing hematopoietic cell transplantation. Thus, given the historically poor survival of hematopoietic cell transplantatio ... Full text Link to item Cite

Non-myeloablative umbilical cord blood transplantation for atypical dyskeratosis congenita.

Journal Article Pediatr Transplant · March 2022 BACKGROUND: Short telomere syndrome (STS) in children may result in phenotypically heterogenous clinical spectrum ranging from completely asymptomatic to typical dyskeratosis congenita (DC). Patients with this cancer predisposition syndrome may have multip ... Full text Link to item Cite

The Role of Granulocyte Transfusions in Optimizing Candidacy for Chimeric Antigen Receptor T-cell Therapy in Patients With Treatment-refractory Infections.

Journal Article J Pediatr Hematol Oncol · March 1, 2022 While advancements in cellular therapy have improved outcomes for patients with refractory leukemia, severe infections may hinder access. Granulocyte transfusions, in combination with anti-microbial therapy, may be a safe option to facilitate candidacy for ... Full text Link to item Cite

Extracorporeal membrane oxygenation in children receiving haematopoietic cell transplantation and immune effector cell therapy: an international and multidisciplinary consensus statement.

Journal Article Lancet Child Adolesc Health · February 2022 Use of extracorporeal membrane oxygenation (ECMO) in children receiving haematopoietic cell transplantation (HCT) and immune effector cell therapy is controversial and evidence-based guidelines have not been established. Remarkable advancements in HCT and ... Full text Link to item Cite

IL12 immune therapy clinical trial review: Novel strategies for avoiding CRS-associated cytokines.

Journal Article Front Immunol · 2022 Interleukin 12 (IL-12) is a naturally occurring cytokine that plays a key role in inducing antitumor immune responses, including induction of antitumor immune memory. Currently, no IL-12-based therapeutic products have been approved for clinical applicatio ... Full text Link to item Cite

Cell membrane-anchored and tumor-targeted IL-12 (attIL12)-T cell therapy for eliminating large and heterogeneous solid tumors.

Journal Article J Immunother Cancer · January 2022 BACKGROUND: Adoptive T-cell transfer has become an attractive therapeutic approach for hematological malignancies but shows poor activity against large and heterogeneous solid tumors. Interleukin-12 (IL-12) exhibits potent antitumor efficacy against solid ... Full text Link to item Cite

Using the MDASI-Adolescent for Early Symptom Identification and Mitigation of Symptom Impact on Daily Living in Adolescent and Young Adult Stem Cell Transplant Patients

Journal Article Children · January 1, 2022 Hematopoietic stem cell transplantation (HSCT) requires an intensive pre-and post-procedure course that leads to symptoms including fatigue, nausea/vomiting, and pain, all of which interfere significantly with activities of daily living. These symptoms pla ... Full text Cite

Venetoclax for children and adolescents with acute lymphoblastic leukemia and lymphoblastic lymphoma

Journal Article Cancers · January 1, 2022 Venetoclax is approved for adult patients with chronic lymphocytic leukemia and acute myeloid leukemia. Expanding its use to the pediatric population is currently under investigation, but more robust data are needed. We retrospectively analyzed the safety ... Full text Cite

Retrospective Review of Flexible Bronchoscopy in Pediatric Cancer Patients

Journal Article Frontiers in Oncology · December 14, 2021 The use of flexible bronchoscopy (FB) with bronchoalveolar lavage (BAL) to diagnose and manage pulmonary complications has been shown to be safe in adult cancer patients, but whether its use is safe in pediatric cancer patients remains unclear. Thus, to de ... Full text Cite

The tki era in chronic leukemias

Journal Article Pharmaceutics · December 1, 2021 Tyrosine kinases are proteins involved in physiological cell functions including proliferation, differentiation, and survival. However, the dysregulation of tyrosine kinase pathways occurs in malignancy, including hematological leukemias such as chronic my ... Full text Cite

Cardiac relapse of acute lymphoblastic leukemia following hematopoietic stem cell transplantation: A case report and review of literature

Journal Article Cancers · November 1, 2021 Isolated extramedullary relapse of acute lymphoblastic leukemia (ALL) occurs in soft tissues and various organs outside the testis and central nervous system. Treatments such as hematopoietic stem cell transplantation and more novel modalities such as immu ... Full text Cite

Early Cumulative Fluid Balance and Outcomes in Pediatric Allogeneic Hematopoietic Cell Transplant Recipients With Acute Respiratory Failure: A Multicenter Study

Journal Article Frontiers in Oncology · July 20, 2021 Objectives: To evaluate the associations between early cumulative fluid balance (CFB) and outcomes among critically ill pediatric allogeneic hematopoietic cell transplant (HCT) recipients with acute respiratory failure, and determine if these associations ... Full text Cite

Diagnosis, grading and management of toxicities from immunotherapies in children, adolescents and young adults with cancer.

Journal Article Nat Rev Clin Oncol · July 2021 Cancer immunotherapies are associated with remarkable therapeutic response rates but also with unique and severe toxicities, which potentially result in rapid deterioration in health. The number of clinical applications for novel immune effector-cell thera ... Full text Link to item Cite

Vitamin D has no impact on outcomes after HSCT in children-A retrospective study.

Journal Article Pediatr Transplant · June 2021 Vitamin D not only plays an important role in bone metabolism but is also involved in multiple immune-mediated processes in the body which may be adversely affected in those with low levels. Most pediatric studies evaluating the association of vitamin D in ... Full text Link to item Cite

Corrigendum: Perspective: A Framework to Screen Pediatric and Adolescent Hematopoietic Cellular Therapy Patients for Organ Dysfunction: Time for a Multi-Disciplinary and Longitudinal Approach (Frontiers in Oncology, (2021), 11, (622630), 10.3389/fonc.2021.622630)

Journal Article Frontiers in Oncology · April 23, 2021 In the original article, there was a mistake in Table 2. Possible Screening for pMODS for Pediatric and Adolescent-Young Adult (AYA) HCT Patients as published. The following in Table 2 has been corrected: Respiratory: Score 0 and Score 2; CV: Score 2; Rena ... Full text Cite

Delirium in Children Undergoing Hematopoietic Cell Transplantation: A Multi-Institutional Point Prevalence Study

Journal Article Frontiers in Oncology · April 22, 2021 Introduction: Delirium occurs frequently in adults undergoing hematopoietic cell transplantation, with significant associated morbidity. Little is known about the burden of delirium in children in the peri-transplant period. This study was designed to dete ... Full text Cite

Lactic Acidosis Secondary to Thiamin Deficiency Following Autologous Stem Cell Transplantation.

Journal Article Nutr Clin Pract · April 2021 A 22-month-old female with high-risk neuroblastoma completed 5 cycles of chemotherapy then underwent high-dose chemotherapy with autologous stem cell rescue (ASCR). Parenteral nutrition was administered from day +2 following ASCR, as she was unable to tole ... Full text Link to item Cite

Immune Effector Cell Associated Neurotoxicity (ICANS) in Pediatric and Young Adult Patients Following Chimeric Antigen Receptor (CAR) T-Cell Therapy: Can We Optimize Early Diagnosis?

Journal Article Frontiers in Oncology · March 8, 2021 The Cornell Assessment for Pediatric Delirium (CAPD) was first proposed by the Pediatric Acute Lung Injury and Sepsis Investigators Network-Stem Cell Transplantation and Cancer Immunotherapy Subgroup and MD Anderson CARTOX joint working committees, for det ... Full text Cite

Perspective: A Framework to Screen Pediatric and Adolescent Hematopoietic Cellular Therapy Patients for Organ Dysfunction: Time for a Multi-Disciplinary and Longitudinal Approach

Journal Article Frontiers in Oncology · February 24, 2021 Hematopoietic Cell Transplantation (HCT) is a potentially curative therapy for children and adolescent/young adults (AYA) with high-risk malignancies as well as some non-malignant genetic diseases. However, HCT may be associated with endotheliopathies and/ ... Full text Cite

Case Discussion and Literature Review: Cancer Immunotherapy, Severe Immune-Related Adverse Events, Multi-Inflammatory Syndrome, and Severe Acute Respiratory Syndrome Coronavirus 2

Journal Article Frontiers in Oncology · February 4, 2021 Pediatric, adolescent and young adult (AYA) patients receiving novel cancer immunotherapies may develop associated toxicities with overlapping signs and symptoms that are not always easily distinguished from severe acute respiratory syndrome coronavirus 2 ... Full text Cite

A pragmatic multi-institutional approach to understanding transplant-associated thrombotic microangiopathy after stem cell transplant.

Journal Article Blood Adv · January 12, 2021 Transplant-associated thrombotic microangiopathy (TA-TMA) is a severe complication of hematopoietic stem cell transplantation (HSCT). A single-center prospective screening study has shown that the incidence of TA-TMA is much higher than prior retrospective ... Full text Link to item Cite

Risk Factors for Noninvasive Ventilation Failure in Children Post-Hematopoietic Cell Transplant.

Journal Article Front Oncol · 2021 RATIONALE: Little is known on the use of noninvasive ventilation (NIPPV) in pediatric hematopoietic cell transplant (HCT) patients. OBJECTIVE: We sought to describe the landscape of NIPPV use and to identify risk factors for failure to inform future invest ... Full text Link to item Cite

Improved detection of sinusoidal obstructive syndrome using pediatric-AYA diagnostic criteria and severity grading.

Journal Article Bone Marrow Transplant · January 2021 New diagnostic criteria and severity grading for sinusoidal obstructive syndrome (SOS) among pediatric and adolescent young adult (AYA) patients have been recently endorsed by international consensus. The extent to which these have been adopted in the US r ... Full text Link to item Cite

Care of the Critically Ill Pediatric Hematopoietic Cell Transplant Patient

Chapter · January 1, 2021 Although potentially curative therapy, hematopoietic cell transplantation (HCT) is fraught with many unique and life-threatening complications. Pulmonary complications, both infectious and noninfectious, are common and a significant source of nonrelapse mo ... Full text Cite

The Organ Trail: A Review of Biomarkers of Organ Failure

Journal Article Frontiers in Oncology · November 11, 2020 Pediatric organ failure and transplant populations face significant risks of morbidity and mortality. The risk of organ failure itself may be disproportionately higher among pediatric oncology patients, as cancer may originate within and/or metastasize to ... Full text Cite

Feasibility and acceptability findings of an energy balance data repository of children, adolescents, and young adults with cancer

Journal Article Journal of Clinical Medicine · September 1, 2020 Cancer patients suffer changes in energy balance (EB), the combination of energy intake (nutrition) and energy expenditure (physical activity (PA)), which may influence cancer-related morbidity, mortality, and quality of life. Significant gaps remain in ou ... Full text Cite

Transfusion reactions in pediatric and adolescent young adult haematology oncology and immune effector cell patients

Journal Article Eclinicalmedicine · September 1, 2020 Background: Active surveillance for transfusion reactions is critically important among pediatric patients undergoing chemotherapy. Among pediatric-adolescent-young-adult (AYA) hematology/oncology patients, who have been typically excluded from transfusion ... Full text Cite

Complete remission in refractory acute lymphoblastic leukemia using blinatumomab after failure of response to CD-19 chimeric antigen receptor T-cell therapy

Journal Article Clinical Case Reports · September 1, 2020 The T-cell engager monoclonal antibody, blinatumomab, is a potential therapeutic strategy for refractory B acute lymphoblastic leukemia after failure of CD 19 chimeric antigen receptor T-cell therapy. ... Full text Cite

Chimeric Antigen Receptor T-Cells in B-Acute Lymphoblastic Leukemia: State of the Art and Future Directions

Journal Article Frontiers in Oncology · August 26, 2020 Use of adoptive T-cell therapy modified with chimeric antigen receptor (CAR-T) has revolutionized treatment of patients with relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL). CAR-T cells directed against CD19 antigen have produced resp ... Full text Cite

Respiratory pathogens associated with intubated pediatric patients following hematopoietic cell transplant.

Journal Article Transpl Infect Dis · August 2020 BACKGROUND: We describe organisms found in the respiratory tracts of a multicenter cohort of pediatric hematopoietic cell transplant (HCT) recipients with respiratory failure. METHODS: Twelve centers contributed up to 25 pediatric allogeneic HCT recipients ... Full text Link to item Cite

A Phase 3, Single-Arm, Prospective Study of Remestemcel-L, Ex Vivo Culture-Expanded Adult Human Mesenchymal Stromal Cells for the Treatment of Pediatric Patients Who Failed to Respond to Steroid Treatment for Acute Graft-versus-Host Disease.

Journal Article Biol Blood Marrow Transplant · May 2020 Steroid-refractory acute graft-versus-host disease (SR-aGVHD) following hematopoietic cell transplantation (HSCT) is associated with poor clinical outcomes. Currently, there are no safe and effective therapies approved for use in the pediatric population u ... Full text Open Access Link to item Cite

Acute respiratory failure and the kinetics of neutrophil recovery in pediatric hematopoietic cell transplantation: a multicenter study.

Journal Article Bone Marrow Transplant · February 2020 In this multicenter study, we investigated the kinetics of neutrophil recovery in relation to acuity and survival among 125 children undergoing allogeneic hematopoietic cell transplantation (allo-HCT) who required invasive mechanical ventilation (IMV). Rec ... Full text Link to item Cite

Correction to: Acute respiratory failure and the kinetics of neutrophil recovery in pediatric hematopoietic cell transplantation: a multicenter study.

Journal Article Bone Marrow Transplant · February 2020 An amendment to this paper has been published and can be accessed via a link at the top of the paper. ... Full text Link to item Cite

Diagnosis, grading, and treatment recommendations for children, adolescents, and young adults with sinusoidal obstructive syndrome: an international expert position statement.

Journal Article Lancet Haematol · January 2020 Sinusoidal obstructive syndrome, also known as hepatic veno-occlusive disease, is a potentially life-threatening complication that occurs in children undergoing haemopoietic stem-cell transplantation (HSCT). Differences in the incidence of genetic predispo ... Full text Link to item Cite

Pediatric Acute Lymphoblastic Leukemia, Version 2.2020, NCCN Clinical Practice Guidelines in Oncology.

Journal Article J Natl Compr Canc Netw · January 2020 Acute lymphoblastic leukemia (ALL) is the most common pediatric malignancy. Advancements in technology that enhance our understanding of the biology of the disease, risk-adapted therapy, and enhanced supportive care have contributed to improved survival ra ... Full text Link to item Cite

Chimeric Antigen Receptor, Teamwork, Education, Assessment, and Management (CAR-TEAM): A Simulation-Based Inter-professional Education (IPE) Intervention for Management of CAR Toxicities.

Journal Article Front Oncol · 2020 Chimeric antigen receptor (CAR) therapies such as tisagenlecleucel, indicated for children and young adults with relapsed and/or refractory CD19+ acute lymphoblastic leukemia (ALL), have been associated with striking treatment outcomes and overall survival ... Full text Link to item Cite

Special Considerations for ICU Management of Patients Receiving CAR Therapy

Chapter · January 1, 2020 Chimeric antigen receptor (CAR) T-cells belong to the class of immune effector cell (IEC) therapies, which have been associated with striking clinical outcomes, particularly among patients with relapsed/refractory hematologic malignancies. Yet, they have a ... Full text Cite

Chimeric antigen receptor (CAR) t-cell therapy in the pediatric critical care

Chapter · October 12, 2019 In 2017, the US Food and Drug Administration (FDA) approved a form of cancer immunotherapy, specifically, a chimeric antigen receptor (CAR), for the treatment of pediatric and young adult patients with refractory CD19-positive B-cell precursor acute lympho ... Full text Cite

Tumor-targeted IL-12 combined with tumor resection yields a survival-favorable immune profile.

Journal Article J Immunother Cancer · June 17, 2019 BACKGROUND: Although accumulated evidence provides a strong scientific premise for using immune profiles to predict survival in patients with cancer, a universal immune profile across tumor types is still lacking, and how to achieve a survival-associated i ... Full text Link to item Cite

Management guidelines for paediatric patients receiving chimeric antigen receptor T cell therapy.

Journal Article Nat Rev Clin Oncol · January 2019 In 2017, an autologous chimeric antigen receptor (CAR) T cell therapy indicated for children and young adults with relapsed and/or refractory CD19+ acute lymphoblastic leukaemia became the first gene therapy to be approved in the USA. This innovative form ... Full text Link to item Cite

Hematopoietic Stem Cell Transplant and Cellular Therapy

Chapter · January 1, 2019 Hematopoietic cell transplantation (HCT) and cellular therapy (CT) are potentially lifesaving treatments for children with malignant and nonmalignant diseases. Advancements in care have led to expanding patient eligibility, with overall reduced morbidity a ... Full text Cite

Acute Respiratory Failure in Pediatric Hematopoietic Cell Transplantation: A Multicenter Study.

Journal Article Crit Care Med · October 2018 OBJECTIVES: Acute respiratory failure is common in pediatric hematopoietic cell transplant recipients and has a high mortality. However, respiratory prognostic markers have not been adequately evaluated for this population. Our objectives are to assess res ... Full text Link to item Cite

High-Frequency Oscillatory Ventilation Use and Severe Pediatric ARDS in the Pediatric Hematopoietic Cell Transplant Recipient.

Journal Article Respir Care · April 2018 INTRODUCTION: The effectiveness of high-frequency oscillatory ventilation (HFOV) in the pediatric hematopoietic cell transplant patient has not been established. We sought to identify current practice patterns of HFOV, investigate parameters during HFOV an ... Full text Link to item Cite

Allogeneic hematopoietic stem cell transplantation is associated with cure and durable remission of late-onset primary isolated central nervous system hemophagocytic lymphohistiocytosis.

Journal Article Pediatr Transplant · February 2018 Primary isolated CNS presentation of HLH is exceedingly rare and typically associated with significant morbidity and mortality. We describe an adolescent patient with late-onset, primary isolated CNS HLH and a compound heterozygous PRF1 mutation (c50delT ( ... Full text Link to item Cite

Consensus Report by the Pediatric Acute Lung Injury and Sepsis Investigators and Pediatric Blood and Marrow Transplantation Consortium Joint Working Committees on Supportive Care Guidelines for Management of Veno-Occlusive Disease in Children and Adolescents, Part 3: Focus on Cardiorespiratory Dysfunction, Infections, Liver Dysfunction, and Delirium.

Journal Article Biol Blood Marrow Transplant · February 2018 Some patients with veno-occlusive disease (VOD) have multiorgan dysfunction, and multiple teams are involved in their daily care in the pediatric intensive care unit. Cardiorespiratory dysfunction is critical in these patients, requiring immediate action. ... Full text Link to item Cite

Consensus Report by the Pediatric Acute Lung Injury and Sepsis Investigators and Pediatric Blood and Marrow Transplant Consortium Joint Working Committees on Supportive Care Guidelines for Management of Veno-Occlusive Disease in Children and Adolescents: Part 2-Focus on Ascites, Fluid and Electrolytes, Renal, and Transfusion Issues.

Journal Article Biol Blood Marrow Transplant · December 2017 Even though hepatic veno-occlusive disease (VOD) is a potentially fatal complication of hematopoietic cell transplantation (HCT), there is paucity of research on the management of associated multiorgan dysfunction. To help provide standardized care for the ... Full text Link to item Cite

Consensus Report by Pediatric Acute Lung Injury and Sepsis Investigators and Pediatric Blood and Marrow Transplantation Consortium Joint Working Committees: Supportive Care Guidelines for Management of Veno-Occlusive Disease in Children and Adolescents, Part 1: Focus on Investigations, Prophylaxis, and Specific Treatment.

Journal Article Biol Blood Marrow Transplant · November 2017 Veno-occlusive disease (VOD) is a common and potentially fatal complication in children undergoing hematopoietic cell transplantation (HCT). It occurs in about one-third of all patients undergoing transplantation and is fatal in 50% of patients with severe ... Full text Link to item Cite

Humoral Immune Reconstitution Kinetics after Allogeneic Hematopoietic Stem Cell Transplantation in Children: A Maturation Block of IgM Memory B Cells May Lead to Impaired Antibody Immune Reconstitution.

Journal Article Biol Blood Marrow Transplant · September 2017 Although T cell immune reconstitution after allogeneic hematopoietic stem cell transplantation (allo-HSCT) has been well studied, long-term B cell immune reconstitution remains less characterized. We evaluated humoral immune reconstitution among 71 pediatr ... Full text Link to item Cite

Pediatric Acute Respiratory Distress Syndrome in Pediatric Allogeneic Hematopoietic Stem Cell Transplants: A Multicenter Study.

Journal Article Pediatr Crit Care Med · April 2017 OBJECTIVE: Immunodeficiency is both a preexisting condition and a risk factor for mortality in pediatric acute respiratory distress syndrome. We describe a series of pediatric allogeneic hematopoietic stem cell transplant patients with pediatric acute resp ... Full text Link to item Cite

5-Azacitidine Monotherapy Followed by Related Haploidentical Hematopoietic Stem Cell Transplantation Achieves Durable Remission in a Pediatric Patient With Acute Undifferentiated Leukemia Refractory to High-Dose Chemotherapy.

Journal Article Pediatr Blood Cancer · June 2016 Patients with acute leukemias of undifferentiated lineage (AUL) generally have guarded prognosis. Here, we describe the first reported pediatric patient with AUL refractory to high-dose chemotherapy who achieved clinical remission with ALL maintenance ther ... Full text Link to item Cite

Invasive Mechanical Ventilation and Mortality in Pediatric Hematopoietic Stem Cell Transplantation: A Multicenter Study.

Journal Article Pediatr Crit Care Med · April 2016 OBJECTIVE: To establish the current respiratory practice patterns in pediatric hematopoietic stem cell transplant patients and investigate their associations with mortality across multiple centers. DESIGN: Retrospective cohort between 2009 and 2014. SETTIN ... Full text Link to item Cite

Durable engraftment and correction of hematological abnormalities in children with congenital amegakaryocytic thrombocytopenia following myeloablative umbilical cord blood transplantation.

Journal Article Pediatr Transplant · November 2015 The use of HSCT is the only potentially curative treatment for CAMT, but access is limited by the availability of suitable donors. We report five consecutive patients with CAMT who received MAC and partially HLA-mismatched, UCBT (unrelated, n = 4). Median ... Full text Open Access Link to item Cite

Unrelated donor hematopoietic stem cell transplantation for the treatment of non-malignant genetic diseases: An alemtuzumab based regimen is associated with cure of clinical disease; earlier clearance of alemtuzumab may be associated with graft rejection.

Journal Article Am J Hematol · November 2015 Hematopoietic stem cell transplantation (HSCT) with matched unrelated donors (MUD), offers potentially curative therapy for patients with non-malignant genetic diseases. In this pilot study conducted from 2006 to 2014, we report the outcomes of 15 patients ... Full text Link to item Cite

Graft versus tumor effect in the brain of a child with recurrent metastatic medulloblastoma.

Journal Article Pediatr Blood Cancer · September 2015 Marrow ablative chemotherapy (MAC) with autologous hematopoietic stem cell transplantation (HSCT) is limited by poor bone marrow reserve after chemotherapy and/or radiotherapy, and the extent of bone/bone marrow disease. We report a child with recurrent me ... Full text Link to item Cite

Low-dose total-body irradiation and alemtuzumab-based reduced-intensity conditioning regimen results in durable engraftment and correction of clinical disease among children with chronic granulomatous disease.

Journal Article Pediatr Transplant · June 2015 HSCT with MAC is associated with durable donor engraftment for patients with CGD; however, MAC is limited by high rates of RRT. We used a novel RIC regimen with LD-TBI (200 cGy × two doses), fludarabine (30 mg/m(2) × three doses), and proximal alemtuzumab ... Full text Link to item Cite

Successful bone marrow transplantation of an adolescent young adult female with pregnancy-associated aplastic anemia.

Journal Article J Pediatr Hematol Oncol · May 2015 Cases of pregnancy-associated severe aplastic anemia (SAA) have been reported in the literature with historically high rates of mortality. We report the case of a 17-year-old female diagnosed with SAA at 26 weeks of gestation. She experienced intrauterine ... Full text Link to item Cite

A survey of infectious disease clinical practices among pediatric blood and marrow transplant programs in the United States.

Journal Article Pediatr Blood Cancer · April 2015 We assessed clinical practice standards for infectious complications among pediatric blood and marrow transplant (PBMT) recipients. An anonymous online survey was sent to all 64 pediatric program directors (PD) of PBMT centers in the United States, which a ... Full text Link to item Cite

A reduced-toxicity regimen is associated with durable engraftment and clinical cure of nonmalignant genetic diseases among children undergoing blood and marrow transplantation with an HLA-matched related donor.

Journal Article Biol Blood Marrow Transplant · March 2015 Blood and marrow transplantation (BMT) is a standard curative therapy for patients with nonmalignant genetic diseases. Myeloablative conditioning has been associated with significant regimen-related toxicity (RRT), whereas reduced-intensity conditioning re ... Full text Link to item Cite

Overcoming psychosocial and developmental barriers to blood and marrow transplantation (BMT) in an adolescent/young adult (AYA) transgender patient with chronic myelogenous leukemia.

Journal Article Pediatr Hematol Oncol · November 2014 Adolescents/young adults (AYAs) afflicted with cancer face unique barriers to potentially standard curative therapies, such as blood and marrow transplantation (BMT). Transgender AYAs face additional barriers and there is a dearth of published literature r ... Full text Link to item Cite

Immunologic resolution of human chronic graft-versus-host disease.

Journal Article Biol Blood Marrow Transplant · October 2014 To determine the role of regulatory T lymphocytes (Tregs) in the pathogenesis of human chronic graft-versus-host disease (GVHD) and its clinical resolution, we evaluated long-term recipients of pediatric allogeneic hematopoietic stem cell transplantation ( ... Full text Link to item Cite

T lymphocyte abnormalities in juvenile systemic sclerosis patients.

Journal Article Clin Immunol · October 2013 Multi-center evaluations of pediatric patients with juvenile systemic sclerosis (jSSc) have suggested that the pathogenesis of jSSc may differ from that of systemic sclerosis (SSc) in adult patients. Therefore, we undertook to identify abnormalities in the ... Full text Link to item Cite

Systemic mastocytosis in a child with t(8;21) acute myeloid leukemia.

Journal Article Pediatr Blood Cancer · October 2011 Mastocytosis is primarily limited to the cutaneous variant in pediatric patients. Systemic mastocytosis (SM) has been associated with t(8;21) acute myeloid leukemia (AML) in adults. We provide the first report of a child with t(8;21) AML, diagnosed with as ... Full text Link to item Cite

Identification of novel mutations in the proton-coupled folate transporter (PCFT-SLC46A1) associated with hereditary folate malabsorption.

Journal Article Mol Genet Metab · May 2011 Hereditary folate malabsorption (HFM) is an autosomal recessive disorder, recently shown to be due to loss-of-function mutations of the proton-coupled folate transporter (PCFT-SLC46A1), resulting in systemic and central nervous system folate deficiency. Da ... Full text Link to item Cite

Prevalence of a loss-of-function mutation in the proton-coupled folate transporter gene (PCFT-SLC46A1) causing hereditary folate malabsorption in Puerto Rico

Journal Article Journal of Pediatrics · January 1, 2011 Objective: To determine whether subjects of Puerto Rican heritage are at increased risk for a specific mutation of the proton-coupled folate transporter (PCFT) causing hereditary folate malabsorption (HFM). Study design: Three percent of the births in Puer ... Full text Cite

Ambulatory high-dose methotrexate administration among pediatric osteosarcoma patients in an urban, underserved setting is feasible, safe, and cost-effective.

Journal Article Pediatr Blood Cancer · December 15, 2010 BACKGROUND: We describe the safety, feasibility, and provide a cost-estimate of outpatient high-dose methotrexate administration (HDMTX) among an urban, underserved population. PROCEDURE: A retrospective analysis of ambulatory HDMTX administration among os ... Full text Link to item Cite

Mutation of the proton-coupled folate transporter gene (PCFT-SLC46A1) in Turkish siblings with hereditary folate malabsorption.

Journal Article Pediatr Hematol Oncol · November 2010 Hereditary folate malabsorption (HFM) is a rare autosomal recessive disorder characterized by systemic and central nervous system folate deficiency. Turkish siblings are reported with the clinical syndrome of HFM, homozygous for deletion of 2 bases (c.204_ ... Full text Link to item Cite

Properties of the Arg376 residue of the proton-coupled folate transporter (PCFT-SLC46A1) and a glutamine mutant causing hereditary folate malabsorption.

Journal Article Am J Physiol Cell Physiol · November 2010 The proton-coupled folate transporter (PCFT-SLC46A1) is required for intestinal folate absorption and is mutated in the autosomal recessive disorder, hereditary folate malabsorption (HFM). This report characterizes properties and requirements of the R376 r ... Full text Link to item Cite

Exacerbation of anthracycline-induced early chronic cardiomyopathy with ATRA: role of B-type natriuretic peptide as an indicator of cardiac dysfunction.

Journal Article J Pediatr Hematol Oncol · March 2010 Cardiac disease is a significant complication of childhood oncologic therapy. We report the case of a 14-year-old female with acute promyelocytic leukemia who developed symptomatic cardiomyopathy only 4 months into treatment with a combination of daunomyci ... Full text Link to item Cite

Subacute methotrexate neurotoxicity and cerebral venous sinus thrombosis in a 12-year-old with acute lymphoblastic leukemia and methylenetetrahydrofolate reductase (MTHFR) C677T polymorphism: homocysteine-mediated methotrexate neurotoxicity via direct endothelial injury.

Journal Article Pediatr Hematol Oncol · February 2010 From as early as the 1970s methotrexate has been associated with disseminated necrotizing leukoencephalopathy and other neurotoxic sequelae. Yet, a clear mechanism for methotrexate-induced neurotoxicity has not been established. The authors describe the ca ... Full text Link to item Cite

The role of protein kinase C in laminin-mediated neurite outgrowth.

Journal Article Biochem Biophys Res Commun · March 5, 1999 Laminin is a potent stimulator of neurite outgrowth in rat pheochromocytoma (PC12) cells. Here, we investigated the role of protein kinase C (PKC) in the mechanism of laminin-mediated neurite outgrowth in PC12 cells. Phorbol ester activators of PKC have be ... Full text Link to item Cite