Abstract |
Myeloablative conditioning (MAC) regimens are commonly used in transplantation for chronic granulomatous disease (CGD) but are associated with toxicity. Reduced-intensity conditioning (RIC) regimens have lower toxicity but may fail to achieve stable donor chimerism. We report a comparison between 4 patients who received a RIC regimen consisting of alemtuzumab (1 mg/kg), fludarabine (150 mg/m2), and melphalan (140 mg/m2) and 14 patients who received a MAC regimen consisting of busulfan (area under the curve, 1800 to 2000 µMol/min twice daily × 4 days), cyclophosphamide (50 mg/kg/day × 4), and antithymocyte globulin (15 mg/kg twice daily on days -2 and -1, then daily on days +1 and +2). Seventy-five percent (n = 3) of RIC patients developed mixed chimerism and needed either withdrawal of immune suppression (n = 1) or additional stem cell products (n = 2) to achieve stable donor chimerism. Ninety-two percent (n = 13) of patients in the MAC group maintained >95% donor chimerism. Complications included acute graft-versus-host disease (MAC 64%, RIC 0%), chronic graft-versus-host disease (MAC 28%, RIC 0%), sinusoidal obstructive syndrome (MAC 7%, RIC 0%), bacteremia (MAC 42%, RIC 0%), fungemia (MAC 14%, RIC 0%), viral disease (MAC 7%, RIC 25%), and death (MAC 21%, RIC 0%). A RIC regimen has lower toxicity but frequently requires interventions to maintain donor chimerism compared with a MAC regimen in CGD.
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Authors | Pooja Khandelwal, Jacob J Bleesing, Stella M Davies, Rebecca A Marsh |
Journal | Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
(Biol Blood Marrow Transplant)
Vol. 22
Issue 11
Pg. 2011-2018
(11 2016)
ISSN: 1523-6536 [Electronic] United States |
PMID | 27543157
(Publication Type: Comparative Study, Journal Article)
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Copyright | Copyright © 2016 The American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved. |
Chemical References |
- Antilymphocyte Serum
- Myeloablative Agonists
- Alemtuzumab
- Cyclophosphamide
- Vidarabine
- Busulfan
- fludarabine
- Melphalan
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Topics |
- Adolescent
- Alemtuzumab
(administration & dosage)
- Antilymphocyte Serum
(administration & dosage)
- Antineoplastic Combined Chemotherapy Protocols
(therapeutic use)
- Busulfan
(administration & dosage)
- Child
- Child, Preschool
- Chimerism
- Cyclophosphamide
(administration & dosage)
- Female
- Granulomatous Disease, Chronic
(complications, therapy)
- Hematopoietic Stem Cell Transplantation
(methods)
- Humans
- Infant
- Male
- Melphalan
(administration & dosage)
- Myeloablative Agonists
(therapeutic use)
- Transplantation Conditioning
(methods)
- Transplantation, Homologous
- Vidarabine
(administration & dosage, analogs & derivatives)
- Young Adult
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