Abstract |
Objective: The outcomes of alternative donor hematopoietic stem cell transplantation (HSCT) with unmanipulated grafts for Inherited bone marrow failure syndromes ( IBMFS) are discouraging. Our study is to demonstrate that IBMFS with disease-specific characteristics requires a tailored conditioning regimens to enhance engraftment and reduce regimen related toxicities. Methods: We retrospectively analyzed 42 patients diagnosed with IBMFS and transplanted with an alternative donor graft at our center from November 2012 to August 2018. Twenty-seven patients had Fanconi anemia (FA), 7 had dyskeratosis congenita (DC), and 8 had severe congenital neutropenia (SCN). Patients received ex-vivo unmanipulated alternative donor grafts from a matched unrelated donor (MUD) (n = 22), haploidentical donor (HID) (n = 17) and unrelated cord blood donor (UCBD) (n = 3). FA and DC patient subgroups received reduce intensified conditioning (RIC), while SCN patients received a myeloablative conditioning ( MAC) regimen. Results: The median follow-up time for the surviving patients was 38 months (range: 9-63 months). The failure-free survival (FFS) for entire cohort was 76.1%, and was 72.4%, 100% and 56.2% for patients with FA, DC and SCN, respectively. There were no primary graft failures. The cumulative incidence of aGVHD at day 100 was 48.1%. The cumulative incidence of cGVHD at 1 and 3 years was 35.0% and 69.3%, respectively. Conclusion: HSCT using alternative donors with unmanipulated grafts and disease-specific conditioning regimens for IBMFS patients shows promising survival.
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Authors | Yue Lu, Min Xiong, Rui-Juan Sun, Yan-Li Zhao, Jian-Ping Zhang, Xing-Yu Cao, De-Yan Liu, Zhi-Jie Wei, Jia-Rui Zhou, Dao-Pei Lu |
Journal | Hematology (Amsterdam, Netherlands)
(Hematology)
Vol. 26
Issue 1
Pg. 134-143
(Dec 2021)
ISSN: 1607-8454 [Electronic] England |
PMID | 33491597
(Publication Type: Journal Article)
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Topics |
- Adolescent
- Adult
- Child
- Child, Preschool
- Congenital Bone Marrow Failure Syndromes
(therapy)
- Female
- Follow-Up Studies
- Graft Survival
- Graft vs Host Disease
(etiology)
- Hematopoietic Stem Cell Transplantation
(adverse effects, methods)
- Humans
- Male
- Survival Analysis
- Transplantation Conditioning
(adverse effects, methods)
- Unrelated Donors
- Young Adult
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