Disease-specific hematopoietic stem cell transplantation in children with inherited bone marrow failure syndromes.

Abstract

Hematopoietic stem cell transplantation (HSCT) using an optimized conditioning regimen is essential for the long-term survival of patients with inherited bone marrow failure syndromes (IBMFS). We report HSCT in 24 children with Fanconi anemia (FA, n = 12), Diamond-Blackfan anemia (DBA, n = 7), and dyskeratosis congenita (DC, n = 5) from a single HSCT center. The graft source was peripheral blood stem cells (n = 19) or cord blood stem cells (n = 5). FA and DC patients received reduced-intensity conditioning, while DBA patients had myeloablative conditioning. The median numbers of infused mononuclear cells and CD34+ cells were 14.20 × 108/kg and 4.3 × 106/kg, respectively. The median time for neutrophil and platelet recovery was 12 and 18 days, respectively. Complete donor engraftment was achieved in 23 of 24 patients. There was one primary graft failure. During a median follow-up of 27.5 months (range, 2-130 months), the overall survival in all patients was 95.8%. The incidence of grade II-III acute graft versus host disease (GvHD) and chronic GvHD was 29.2% and 16.7%, respectively. We conclude that HSCT can be a curative option for patients with IBMFS. Modification of the conditioning regimen based on the type of disease may lead to encouraging long-term outcomes.

• Authors: Qian Li, Changying Luo, Chengjuan Luo, Jianmin Wang, Benshang Li, Lixia Ding, Jing Chen
• Journal: Annals of hematology (Ann Hematol) Vol. 96 Issue 8 Pg. 1389-1397 (Aug 2017) ISSN: 1432-0584 [Electronic] Germany
• PMID: 28623394 (Publication Type: Journal Article)

Topics

• Adolescent
• Anemia, Aplastic (therapy)
• Anemia, Diamond-Blackfan (therapy)
• Bone Marrow Diseases (therapy)
• Bone Marrow Failure Disorders
• Child
• Child, Preschool
• Cord Blood Stem Cell Transplantation (adverse effects, methods)
• Donor Selection
• Dyskeratosis Congenita (therapy)
• Fanconi Anemia (therapy)
• Female
• Graft vs Host Disease (diagnosis, etiology)
• Hematopoietic Stem Cell Transplantation (adverse effects, methods)
• Hematuria (diagnosis, etiology)
• Hemoglobinuria, Paroxysmal (therapy)
• Humans
• Infant
• Kaplan-Meier Estimate
• Male
• Outcome Assessment, Health Care
• Peripheral Blood Stem Cell Transplantation (adverse effects, methods)
• Transplantation Conditioning