Haploidentical Hematopoietic Cell Transplantation with Post-Transplant Cyclophosphamide in a Patient with Chronic Granulomatous Disease and Active Infection: A First Report

Purpose We describe haploidentical hematopoietic cell transplantation (HCT) with high-dose post-transplant cyclophosphamide (PTCy) in a boy with x-linked chronic granulomatous disease (CGD). Methods A persistent and life-threatening fungal infection was the indication for HSCT. Non-myeloablative con...

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Bibliographic Details
Published in:Journal of clinical immunology 2015-10, Vol.35 (7), p.675-680
Main Authors: Parta, Mark, Hilligoss, Dianne, Kelly, Corin, Kwatemaa, Nana, Theobald, Narda, Malech, Harry, Kang, Elizabeth M.
Format: Article
Language:English
Subjects:
Biomedical and Life Sciences
Biomedicine
Blood Transfusion, Autologous
Chimerism - drug effects
Cyclophosphamide - administration & dosage
Cytomegalovirus
Drug Dosage Calculations
Graft vs Host Disease - etiology
Graft vs Host Disease - prevention & control
Granulomatous Disease, Chronic - complications
Granulomatous Disease, Chronic - therapy
HLA Antigens - immunology
Humans
Immunology
Immunosuppressive Agents - administration & dosage
Infant
Infectious Diseases
Internal Medicine
Male
Medical Microbiology
Mycoses - etiology
Mycoses - therapy
National Institutes of Health (U.S.)
Original Article
Postoperative Complications - prevention & control
Scedosporium
Transplantation Conditioning
United States
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Summary:Purpose We describe haploidentical hematopoietic cell transplantation (HCT) with high-dose post-transplant cyclophosphamide (PTCy) in a boy with x-linked chronic granulomatous disease (CGD). Methods A persistent and life-threatening fungal infection was the indication for HSCT. Non-myeloablative conditioning with PTCy (50 mg/kg days 3 and 4) was used in the absence of fully matched donors. Results Engraftment occurred on day 24. The patient experienced Grade 2 graft-versus-host disease of the skin and gastrointestinal tract and CMV infection, both of which were controlled. Chimerism was 100 % at days 30 and 6 months. Cessation of antifungal therapy was consistent with cure of the infection. Conclusions Haploidentical HCT with high-dose PTCy for CGD is feasible and succeeded even in the context of active infection.
ISSN:0271-9142
1573-2592
1573-2592
DOI:10.1007/s10875-015-0204-y