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Haploidentical transplant for paediatric patients with severe thalassaemia using post-transplant cyclophosphamide and methotrexate: A prospectively registered multicentre trial from the Bone Marrow Failure Working Group of Hunan Province, China.
- Source :
-
British journal of haematology [Br J Haematol] 2023 Feb; Vol. 200 (3), pp. 329-337. Date of Electronic Publication: 2022 Oct 18. - Publication Year :
- 2023
-
Abstract
- Haploidentical transplantation strategies for patients with transfusion-dependent thalassaemia (TD-TM) remain to be investigated. In this study, 54 paediatric patients with TD-TM were treated with a novel approach using post-transplant cyclophosphamide (PTCy) and low-dose methotrexate (LD-MTX), following a myeloablative regimen. The incidence of neutrophil and platelet engraftment was 96.3% ± 2.6% and 94.4% ± 3.1% respectively. The cumulative incidence of grades II-III acute graft-versus-host disease (GVHD) was 13.8% ± 4.8% at 100 days. At three years, the cumulative incidence of chronic GVHD was 28.5% ± 8.5%. With a median follow-up of 520 days (132-1325 days), the overall survival (OS) and event-free survival (EFS) were 98.1% ± 1.8% and 90.7% ± 3.9% respectively. Compared with the low-dose cyclophosphamide (CTX) conditioning regimen (120 mg/kg), the high-CTX regimen (200 mg/kg) achieved a higher incidence of stable engraftment (100% vs 66.7% ± 15.7%, p = 0.003), a comparable incidence of grades II-III acute GVHD, a lower incidence of chronic GVHD (20.2% ± 8.3% vs 66.6% ± 19.2%, p = 0.011), and better overall survival (100% vs 88.9% ± 10.5%, p = 0.025) as well as EFS (95.6% ± 3.1% vs 66.7% ± 15.7%, p = 0.008). Our results using unmanipulated haploidentical grafts and PTCy with LD-MTX in TD-TM are encouraging. (chictr.org.cn ChiCTR1800017969).<br /> (© 2022 British Society for Haematology and John Wiley & Sons Ltd.)
- Subjects :
- Humans
Child
Methotrexate therapeutic use
Transplantation, Haploidentical adverse effects
Cyclophosphamide therapeutic use
Transplantation Conditioning adverse effects
China
Bone Marrow Failure Disorders drug therapy
Hematopoietic Stem Cell Transplantation adverse effects
Graft vs Host Disease etiology
Graft vs Host Disease prevention & control
Graft vs Host Disease drug therapy
Pancytopenia etiology
Thalassemia complications
Bronchiolitis Obliterans Syndrome
Subjects
Details
- Language :
- English
- ISSN :
- 1365-2141
- Volume :
- 200
- Issue :
- 3
- Database :
- MEDLINE
- Journal :
- British journal of haematology
- Publication Type :
- Academic Journal
- Accession number :
- 36254684
- Full Text :
- https://doi.org/10.1111/bjh.18520