Skip to main content
Journal cover image

Hematopoietic Stem Cell Transplantation for CD40 Ligand Deficiency: Single Institution Experience.

Publication ,  Journal Article
Allewelt, H; Martin, PL; Szabolcs, P; Chao, N; Buckley, R; Parikh, S
Published in: Pediatr Blood Cancer
December 2015

BACKGROUND: X-linked hyper-IgM syndrome (X-HIGM) due to mutations in the gene encoding CD40 ligand results in failure of Ig class switching and an increased propensity for recurrent sinopulmonary and other infections, and thus decreased life expectancy. Allogeneic hematopoietic stem cell transplantation (HSCT) is curative, but long-term follow-up data are limited. PROCEDURES: We conducted a retrospective analysis of seven patients who have undergone allogeneic HSCT for HIGM syndrome at Duke University Medical Center. RESULTS: Median age at transplant was 5.2 years (range 0.7-19.3). None of the patients had active hepatic or pulmonary disease immediately prior to transplant, but all had a history of serious infections. Five patients received myeloablative conditioning, and two patients received reduced intensity conditioning. Graft sources included bone marrow, peripheral blood, and unrelated umbilical cord blood. Post-transplantation complications included veno-occlusive disease, hemorrhagic cystitis, adenoviremia, and cryptosporidium recurrence in one patient each. Two patients developed acute GVHD grades II-IV that resolved promptly with treatment and none developed extensive chronic GVHD. All patients are intravenous IgG-independent and 6/7 have normal antibody titers. Immunoglobulin (Ig) A levels normalized in all but one patient and T and B cell numbers and function are otherwise normal in all. All patients are alive at a median follow-up of 9.7 (range 9.7-16.1) years post-transplantation with predominantly donor chimerism and no recurrent infections. CONCLUSIONS: Allogeneic HSCT results in excellent survival and sustained immune reconstitution in patients with CD40 ligand deficiency using both myeloablative and reduced intensity conditioning approaches and various graft sources, including bone marrow, peripheral blood, and umbilical cord blood.

Duke Scholars

Altmetric Attention Stats
Dimensions Citation Stats

Published In

Pediatr Blood Cancer

DOI

EISSN

1545-5017

Publication Date

December 2015

Volume

62

Issue

12

Start / End Page

2216 / 2222

Location

United States

Related Subject Headings

  • Transplantation Conditioning
  • Retrospective Studies
  • Recovery of Function
  • Pulmonary Veno-Occlusive Disease
  • Oncology & Carcinogenesis
  • Male
  • Infant
  • Immunoglobulins, Intravenous
  • Hyper-IgM Immunodeficiency Syndrome, Type 1
  • Humans
 

Citation

APA
Chicago
ICMJE
MLA
NLM
Allewelt, H., Martin, P. L., Szabolcs, P., Chao, N., Buckley, R., & Parikh, S. (2015). Hematopoietic Stem Cell Transplantation for CD40 Ligand Deficiency: Single Institution Experience. Pediatr Blood Cancer, 62(12), 2216–2222. https://doi.org/10.1002/pbc.25711
Allewelt, Heather, Paul L. Martin, Paul Szabolcs, Nelson Chao, Rebecca Buckley, and Suhag Parikh. “Hematopoietic Stem Cell Transplantation for CD40 Ligand Deficiency: Single Institution Experience.Pediatr Blood Cancer 62, no. 12 (December 2015): 2216–22. https://doi.org/10.1002/pbc.25711.
Allewelt H, Martin PL, Szabolcs P, Chao N, Buckley R, Parikh S. Hematopoietic Stem Cell Transplantation for CD40 Ligand Deficiency: Single Institution Experience. Pediatr Blood Cancer. 2015 Dec;62(12):2216–22.
Allewelt, Heather, et al. “Hematopoietic Stem Cell Transplantation for CD40 Ligand Deficiency: Single Institution Experience.Pediatr Blood Cancer, vol. 62, no. 12, Dec. 2015, pp. 2216–22. Pubmed, doi:10.1002/pbc.25711.
Allewelt H, Martin PL, Szabolcs P, Chao N, Buckley R, Parikh S. Hematopoietic Stem Cell Transplantation for CD40 Ligand Deficiency: Single Institution Experience. Pediatr Blood Cancer. 2015 Dec;62(12):2216–2222.
Journal cover image

Published In

Pediatr Blood Cancer

DOI

EISSN

1545-5017

Publication Date

December 2015

Volume

62

Issue

12

Start / End Page

2216 / 2222

Location

United States

Related Subject Headings

  • Transplantation Conditioning
  • Retrospective Studies
  • Recovery of Function
  • Pulmonary Veno-Occlusive Disease
  • Oncology & Carcinogenesis
  • Male
  • Infant
  • Immunoglobulins, Intravenous
  • Hyper-IgM Immunodeficiency Syndrome, Type 1
  • Humans