Scholars@Duke
Timothy Alan Driscoll

Timothy Alan Driscoll

Associate Professor of Pediatrics
Pediatrics, Transplant and Cellular Therapy
timothy.driscoll@duke.edu
Box 102502 Med Ctr, Durham, NC 27710
2400 Pratt Street, 8th Floor North Pavilion Office# 8043, Durham, NC 27705

Scholarly Works - Journal articles

Autologous stem cell transplant for severe, progressive juvenile systemic sclerosis.

Journal article Stem Cells Transl Med · March 23, 2026 Juvenile systemic sclerosis (jSSc) is a rare, chronic, autoimmune disease in children/adolescents and is associated with significant morbidity, skin thickening/hardening (scleroderma), organ toxicity and sub-optimal therapeutic options. In this report, aut ... Full text Link to item Cite

Real-world data for tisagenlecleucel in patients with R/R B-ALL: subgroup analyses from the CIBMTR registry.

Journal article Blood Adv · October 28, 2025 Since the first approval of tisagenlecleucel in 2017, pediatric and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL) may receive this CD19-directed chimeric antigen receptor T-cell therapy. We report real-world data ... 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

Mediport use as an acceptable standard for CAR T cell infusion.

Journal article Front Immunol · 2023 INTRODUCTION: Mediport use as a clinical option for the administration of chimeric antigen receptor T cell (CAR T cell) therapy in patients with B-cell malignancies has yet to be standardized. Concern for mediport dislodgement, cell infiltration, and ineff ... Full text Link to item Cite

Ruxolitinib for the treatment of steroid refractory pediatric chronic graft-versus-host disease.

Journal article Pediatr Dermatol · May 2022 Chronic graft-versus-host disease (cGVHD) of the skin is a serious cause of long-term morbidity and mortality among patients who receive hematopoietic stem cell transplants. Systemic corticosteroids remain first-line treatment for cutaneous cGVHD; however, ... Full text Link to item Cite

Next-Generation Sequencing of Minimal Residual Disease for Predicting Relapse after Tisagenlecleucel in Children and Young Adults with Acute Lymphoblastic Leukemia.

Journal article Blood Cancer Discov · January 2022 We assessed minimal residual disease (MRD) detection and B-cell aplasia after tisagenlecleucel therapy for acute lymphoblastic leukemia (ALL) to define biomarkers predictive of relapse (N = 143). Next-generation sequencing (NGS) MRD detection >0 in bone ma ... Full text Link to item Cite

Non-inferiority of fragmented care for high-risk pediatric neuroblastoma patients: a single institution analysis.

Journal article Pediatr Hematol Oncol · November 2021 Pediatric neuroblastoma (NB) patients receive multi-modal therapy and may experience care fragmented among multiple institutions with a significant travel burden, which has been associated with poor outcomes for some adult cancers. We hypothesized that fra ... Full text Link to item Cite

Pooled safety analysis of tisagenlecleucel in children and young adults with B cell acute lymphoblastic leukemia.

Journal article J Immunother Cancer · August 2021 BACKGROUND: Tisagenlecleucel, an anti-CD19 chimeric antigen receptor T cell therapy, has demonstrated efficacy in children and young adults with relapsed/refractory B cell acute lymphoblastic leukemia (B-ALL) in two multicenter phase 2 trials (ClinicalTria ... Full text Link to item Cite

Patient-reported quality of life after tisagenlecleucel infusion in children and young adults with relapsed or refractory B-cell acute lymphoblastic leukaemia: a global, single-arm, phase 2 trial.

Journal article Lancet Oncol · December 2019 BACKGROUND: The ELIANA trial showed that 61 (81%) of 75 paediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukaemia achieved overall remission after treatment with tisagenlecleucel, a chimeric antigen receptor targe ... Full text Link to item Cite

Massive clonal expansion of medulloblastoma-specific T cells during adoptive cellular therapy.

Journal article Sci Adv · November 2019 In both human and murine systems, we have developed an adoptive cellular therapy platform against medulloblastoma and glioblastoma that uses dendritic cells pulsed with a tumor RNA transcriptome to expand polyclonal tumor-reactive T cells against a plurali ... Full text Open Access Link to item Cite

Reduction in Mortality after Umbilical Cord Blood Transplantation in Children Over a 20-Year Period (1995-2014).

Journal article Biol Blood Marrow Transplant · April 2019 Infections and graft-versus-host disease (GVHD) have historically resulted in high mortality among children undergoing umbilical cord blood transplantation (UCBT). However, recent advances in clinical practice have likely improved outcomes of these patient ... Full text Open Access Link to item Cite

Ethical considerations of using a single minor donor for three bone marrow harvests for three HLA-matched siblings with primary immunodeficiency.

Journal article Pediatr Blood Cancer · April 2019 Allogeneic hematopoietic stem cell transplantation is curative for primary immunodeficiencies. Bone marrow from an unaffected human leukocyte antigen (HLA)-identical sibling donor is the ideal graft source. For minor donors, meaningful consent or assent ma ... Full text Open Access Link to item Cite

Durable Chimerism and Long-Term Survival after Unrelated Umbilical Cord Blood Transplantation for Pediatric Hemophagocytic Lymphohistiocytosis: A Single-Center Experience.

Journal article Biol Blood Marrow Transplant · October 2017 Hemophagocytic lymphohistiocytosis (HLH) is a life-threatening disorder of immune dysregulation characterized by fever, hepatosplenomegaly, cytopenias, central nervous system disease, increased inflammatory markers, and hemophagocytosis. Currently, allogen ... Full text Open Access Link to item Cite

Children Receiving Hematopoietic Stem Cell Transplantation are at Increased Risk of Onychocryptosis Requiring Surgical Management.

Journal article J Pediatr Hematol Oncol · October 2017 PURPOSE: At our institution, a high proportion of children with onychocryptosis (ingrown toenail) requiring surgical intervention were noted to have a history of hematopoietic stem cell transplantation (HSCT). We analyzed the characteristics of patients wh ... 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

Gorlin syndrome and desmoplastic medulloblastoma: Report of 3 cases with unfavorable clinical course and novel mutations.

Journal article Pediatr Blood Cancer · October 2015 We present three cases of genetically confirmed Gorlin syndrome with desmoplastic medulloblastoma (DMB) in whom tumor recurred despite standard therapy. One patient was found to have a novel germline missense PTCH1 mutation. Molecular analysis of recurrent ... Full text Link to item Cite

Ex vivo generation of dendritic cells from cryopreserved, post-induction chemotherapy, mobilized leukapheresis from pediatric patients with medulloblastoma.

Journal article J Neurooncol · October 2015 Generation of patient-derived, autologous dendritic cells (DCs) is a critical component of cancer immunotherapy with ex vivo-generated, tumor antigen-loaded DCs. An important factor in the ability to generate DCs is the potential impact of prior therapies ... Full text Link to item Cite

Value of surgical resection in children with high-risk neuroblastoma.

Journal article Pediatr Blood Cancer · September 2015 BACKGROUND: The value of gross total resection (GTR) for children with high-risk neuroblastoma (NB) is controversial. We hypothesized that patients undergoing GTR would demonstrate improved overall survival (OS) compared those having Full text Link to item Cite

A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with nonmalignant diseases.

Journal article Biol Blood Marrow Transplant · March 2014 Reduced-intensity conditioning (RIC) regimens have the potential to decrease transplantation-related morbidity and mortality. However, engraftment failure has been prohibitively high after RIC unrelated umbilical cord blood transplantation (UCBT) in chemot ... Full text Open Access Link to item Cite

Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning.

Journal article Blood · May 9, 2013 We report transplantation outcomes of 258 children with Hurler syndrome (HS) after a myeloablative conditioning regimen from 1995 to 2007. Median age at transplant was 16.7 months and median follow-up was 57 months. The cumulative incidence of neutrophil r ... Full text Open Access Link to item Cite

Myeloablative transplantation using either cord blood or bone marrow leads to immune recovery, high long-term donor chimerism and excellent survival in chronic granulomatous disease.

Journal article Biol Blood Marrow Transplant · September 2012 The curative potential of hematopoietic stem cell transplantation in patients with chronic granulomatous disease depends on availability of a suitable donor, successful donor engraftment, and maintenance of long-term donor chimerism. Twelve consecutive chi ... Full text Open Access Link to item Cite

Therapeutic monitoring of voriconazole in children less than three years of age: a case report and summary of voriconazole concentrations for ten children.

Journal article Pediatr Infect Dis J · June 2012 Voriconazole is the treatment of choice for invasive aspergillosis and its use is increasing in pediatrics. Minimal pharmacokinetic data exist in young children. We report voriconazole concentrations for 10 children <3 years of age and pharmacokinetic para ... Full text Link to item Cite

Comparison of pharmacokinetics and safety of voriconazole intravenous-to-oral switch in immunocompromised adolescents and healthy adults.

Journal article Antimicrob Agents Chemother · December 2011 The current voriconazole dosing recommendation in adolescents is based on limited efficacy and pharmacokinetic data. To confirm the appropriateness of dosing adolescents like adults, a pharmacokinetic study was conducted in 26 immunocompromised adolescents ... Full text Link to item Cite

Comparison of pharmacokinetics and safety of voriconazole intravenous-to-oral switch in immunocompromised children and healthy adults.

Journal article Antimicrob Agents Chemother · December 2011 Voriconazole pharmacokinetics are not well characterized in children despite prior studies. To assess the appropriate pediatric dosing, a study was conducted in 40 immunocompromised children aged 2 to <12 years to evaluate the pharmacokinetics and safety o ... Full text Link to item Cite

BACPTDP: a water-soluble camptothecin pro-drug with enhanced activity in hypoxic/acidic tumors.

Journal article Cancer Chemother Pharmacol · April 2011 UNLABELLED: Hypoxia is a common feature of solid tumors. Up-regulation of hypoxia-inducing factor-1 (HIF-1) occurs in the majority of primary malignant tumors and in two-thirds of metastases, while most normal tissues are negative. HIF-1 induces the glycol ... Full text Link to item Cite

Pharmacokinetics, safety, and tolerability of voriconazole in immunocompromised children.

Journal article Antimicrob Agents Chemother · October 2010 The pharmacokinetics of voriconazole in children receiving 4 mg/kg intravenously (i.v.) demonstrate substantially lower plasma exposures (as defined by area under the concentration-time curve [AUC]) than those in adults receiving the same therapeutic dosag ... Full text Link to item Cite

Unrelated donor umbilical cord blood transplantation in pediatric myelodysplastic syndrome: a single-center experience.

Journal article Biol Blood Marrow Transplant · August 2009 Myelodysplastic syndromes (MDS) respond poorly to chemotherapy. Between 1995 and 2006, 23 pediatric patients with MDS were transplanted with unrelated donor umbilical cord blood (UUCB) at our center. The median age was 11.1 years (range: 1.1-19.7), median ... Full text Link to item Cite

Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes.

Journal article Blood · October 1, 2008 Outcomes of 159 young patients with inherited metabolic disorders (IMDs) undergoing transplantation with partially HLA-mismatched unrelated donor umbilical cord blood were studied to investigate the impact of graft and patient characteristics on engraftmen ... Full text Link to item Cite

Efficacy of high-dose chemotherapy or standard salvage therapy in patients with recurrent medulloblastoma.

Journal article Neuro Oncol · October 2008 The efficacy of high-dose chemotherapy (HDC) or standard salvage therapy was evaluated in patients with recurrent medulloblastoma (MBL) using retrospective chart review of all patients with recurrent MBL treated at Duke University Medical Center between 19 ... Full text Link to item Cite

Correction of chronic granulomatous disease after second unrelated-donor umbilical cord blood transplantation.

Journal article Pediatr Blood Cancer · December 2007 Allogeneic hematopoietic stem cell transplantation (HSCT) is curative for chronic granulomatous disease (CGD), but many patients lack a suitably matched related donor. We report successful outcomes after mismatched, unrelated-donor umbilical cord blood tra ... Full text Link to item Cite

Prospective Aspergillus galactomannan antigen testing in pediatric hematopoietic stem cell transplant recipients.

Journal article Pediatr Infect Dis J · July 2007 BACKGROUND: The galactomannan (GM) assay is an approved noninvasive test for detection of invasive aspergillosis (IA) that has been validated in adult patients with hematologic malignancies who are undergoing bone marrow transplantation. There have been fe ... Full text Link to item Cite

Outcomes of unrelated umbilical cord blood transplantation for X-linked adrenoleukodystrophy.

Journal article Biol Blood Marrow Transplant · June 2007 Adrenoleukodystrophy (ALD) is an X-linked disorder caused by a defect in the metabolism of long chain fatty acids leading to demyelination, neurodegeneration, and death. The disease typically presents in young boys and adolescent boys. Allogeneic bone marr ... Full text Link to item Cite

Safety and pharmacokinetics of intravenous anidulafungin in children with neutropenia at high risk for invasive fungal infections.

Journal article Antimicrob Agents Chemother · February 2006 Anidulafungin is an echinocandin with activity against Candida species and Aspergillus species. Adult dosages under study are 50 mg/day for esophageal candidiasis and 100 mg/day for invasive candidiasis and aspergillosis. Little is known, however, about th ... Full text Link to item Cite

Improved outcome in central nervous system aspergillosis, using voriconazole treatment.

Journal article Blood · October 15, 2005 The mortality of central nervous system (CNS) aspergillosis approaches 100%, requiring improved therapies. Voriconazole gives superior efficacy and survival in invasive aspergillosis, compared with amphotericin B. Also, in contrast to other antifungal drug ... Full text Link to item Cite

Pharmacokinetics and safety of intravenous voriconazole in children after single- or multiple-dose administration.

Journal article Antimicrob Agents Chemother · June 2004 We conducted a multicenter study of the safety, tolerability, and plasma pharmacokinetics of the parenteral formulation of voriconazole in immunocompromised pediatric patients (2 to 11 years old). Single doses of 3 or 4 mg/kg of body weight were administer ... Full text Link to item Cite

The activity of camptothecin analogues is enhanced in histocultures of human tumors and human tumor xenografts by modulation of extracellular pH.

Journal article Cancer Chemother Pharmacol · September 2003 BACKGROUND: Most solid human tumors exist in an acidic microenvironment, due in part to inefficient vasculature and a higher intrinsic rate of glycolysis. This leads to a tumor-selective pH gradient, which can be exploited therapeutically with antitumor ag ... Full text Link to item Cite

Augmentation of umbilical cord blood (UCB) transplantation with ex vivo-expanded UCB cells: results of a phase 1 trial using the AastromReplicell System.

Journal article Blood · June 15, 2003 Allogeneic stem cell transplantation with umbilical cord blood (UCB) cells is limited by the cell dose a single unit provides recipients. Ex vivo expansion is one strategy to increase the number of cells available for transplantation. Aastrom Biosciences d ... Full text Link to item Cite

Voriconazole in the treatment of aspergillosis, scedosporiosis and other invasive fungal infections in children.

Journal article Pediatr Infect Dis J · March 2002 OBJECTIVE: To describe the safety and efficacy of voriconazole in children treated within the compassionate release program. METHODS: Children received voriconazole on a compassionate basis for treatment of an invasive fungal infection if they were refract ... Full text Link to item Cite

Ceramide generation by two distinct pathways in tumor necrosis factor alpha-induced cell death.

Journal article FEBS Lett · August 10, 2001 Ceramide accumulation in the cell can occur from either hydrolysis of sphingomyelin or by de novo synthesis. In this study, we found that blocking de novo ceramide synthesis significantly inhibits ceramide accumulation and subsequent cell death in response ... Full text Link to item Cite

Graft-versus-leukemia-induced complete remission following unrelated umbilical cord blood transplantation for acute leukemia.

Journal article Bone Marrow Transplant · December 2000 A 15-year-old female received an unrelated three of six HLA antigen matched umbilical cord blood (UCB) transplant for refractory, relapsed T-cell ALL. Conditioning consisted of TBI, melphalan, and anti-thymocyte globulin (ATG), with cyclosporin A (CsA) and ... Full text Link to item Cite

High dose chemotherapy followed by transplantation of unrelated, banked, umbilical cord blood provides curative therapy for young pediatric patients with familial erythrophagocytic lymphohistiocytosis fed

Journal article Blood · December 1, 2000 Familial erythrophagocytic lymphohistiocytosis (PEL) is an inherited disease that is uniformly fatal without myeloablation and transplantation therapy. Frequently babies and young children with this condition need to proceed to transplant rapidly before di ... Cite

Regulation of membrane release in apoptosis.

Journal article Biochem J · September 1, 1998 Apoptosis is a fundamental process of cell regulation whereby cells execute one or more biochemical programs leading to cell death. Several mechanisms have been evaluated and suggested to play roles in the regulation of apoptosis, including the activation ... Full text Link to item Cite