Allogeneic hematopoietic cell transplantation for severe aplastic anemia: similar long-term overall survival after transplantation with related donors compared to unrelated donors

Mortensen, Bo; Jacobsen, Niels; Heilmann, C; Sengeløv, Henrik

doi:10.1038/bmt.2015.231

Cited by 15 publications

(16 citation statements)

References 9 publications

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“…Our prior study suggested the noninferiority of MUDs to MSDs when the pretransplant conditions were well matched in this high-resolution human leukocyte antigen (HLA) typing era, and several studies have supported our analysis [1, 4, 5]. Therefore, alloHSCT from MUDs and haplo-identical family donors is a plausible strategy for severe AA patients without an MSD [6, 7].…”

Section: Introductionsupporting

confidence: 52%

Effect of Stem Cell Source and Dose on Allogeneic Hematopoietic Stem Cell Transplantation in Adult Patients with Idiopathic Aplastic Anemia: Data from the Korean Aplastic Anemia Trials

et al. 2019

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Objective: We aimed to evaluate the effect of stem cell source and dose on the survival of various donor subgroups, such as matched sibling donor (MSDs) and alternative donors (ADs), upon bone marrow (BM) or peripheral blood stem cell (PBSC) infusion in aplastic anemia (AA). Methods: We retrospectively investigated the effects of stem cell source and dose on allogeneic hematopoietic stem cell transplantation (alloHSCT) in AA. Results: A total of 267 patients were included in this analysis. The BM-treated group showed an association with low incidence of any-grade acute graft versus host disease (GvHD) (p < 0.001). A higher stem cell dose was related with a low incidence of extensive chronic GvHD in MSDs (p = 0.025). Multivariate analysis for overall survival (OS) revealed that only age at alloHSCT <31 years (p = 0.010) and prior platelet transfusion <86 U (p = 0.046) in MSDs and higher stem cell dose (hazard ratio = 2.596, p = 0.045) in ADs were favorable prognostic factors. Conclusion: PBSCs could be preferred in AD because high stem cell dose may be easily achieved to improve the OS at the expense of acute GvHD. However, BM stem cells are preferred in MSDs.

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Section: Introductionsupporting

confidence: 52%

Effect of Stem Cell Source and Dose on Allogeneic Hematopoietic Stem Cell Transplantation in Adult Patients with Idiopathic Aplastic Anemia: Data from the Korean Aplastic Anemia Trials

et al. 2019

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“…Moreover, the outcomes after allo-HSCT from an URD have steadily improved over the past three decades. Recent data have revealed similar outcomes for upfront-unrelated and matched sibling HSCT for pediatric AA, which supports the recommendation for first-line treatment with an URD-HSCT for children who lack an MSD [8,9]. In the past, alternative donor HSCT was another salvage choice for cure in patients with refractory AA after IST, but morbidity and mortality from graft failure and complications of graft-versus-host disease (GVHD) have limited clinical applications for this approach.…”

mentioning

confidence: 76%

Allo-HSCT compared with immunosuppressive therapy for acquired aplastic anemia: a system review and meta-analysis

Zhu

Gao

et al. 2020

BMC Immunol

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Background: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) and immunosuppressive therapy (IST) are two major competing treatment strategies for acquired aplastic anemia (AA). Whether allo-HSCT is superior to IST as a front-line treatment for patients with AA has been a subject of debate. To compare the efficacy and safety of allo-HSCT with that of IST as a front-line treatment for patients with AA, we performed a meta-analysis of available studies that examined the impact of the two major competing treatment strategies for AA. Results: Fifteen studies including a total of 5336 patients were included in the meta-analysis. The pooled hazard ratio (HR) for overall survival (OS) was 0.4 (95% CI 0.074-0.733, P = 0.016, I 2 = 58.8%) and the pooled HR for failurefree survival (FFS) was 1.962 (95% CI 1.43-2.493, P = 0.000, I 2 = 0%). The pooled relative risk (RR) for overall response rate (ORR) was 1.691 (95% CI 1.433-1.996, P = 0.000, I 2 = 11.6%). Conclusion: Although survival was significantly longer among AA patients undergoing first-line allo-HSCT compared to those undergoing first-line IST, the selection of initial treatment for patients with newly diagnosed AA still requires comprehensive evaluation of donor availability, patient age, expected quality of life, risk of disease relapse or clonal evolution after IST, and potential use of adjunctive eltrombopag.

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“…BMT from a matched URD represents an alternative treatment strategy for IST‐refractory patients, as well as for children without an available MRD. OS after URD BMT has improved over the past decades in developed countries, and recent studies have observed excellent outcomes, approaching those reported after MRD transplants . Thus, BMT can be considered as first‐line treatment for children with SAA who lack an MRD in developed countries.…”

Section: Introductionmentioning

confidence: 92%

“…OS after URD BMT has improved over the past decades in developed countries, 9,10 and recent studies have observed excellent outcomes, approaching those reported after MRD transplants. [11][12][13] Thus, BMT can be considered as first-line treatment for children with SAA who lack an MRD in developed countries. Transplants from alternative donors (ie, adult mismatched unrelated donors, unrelated cord blood, and haploidentical donors) have also been reported, but with inferior results compared with transplants from MRD.…”

Section: Introductionmentioning

confidence: 99%

Allogeneic Bone Marrow Transplants for Pediatric Severe Aplastic Anemia: Real‐world Data comparing Matched Related and Unrelated Donors in a Developing Country. Retrospective study on behalf of the Pediatric Hematopoietic Stem Cell Transplant Working Group of the Brazilian Bone Marrow Transplantation Society (SBTMO) and the Brazil‐Seattle Consortium (Gedeco)

Darrigo

Colturato

Souza

et al. 2019

Pediatric Transplantation

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In this study, we report on major MRD or URD BMT outcomes in pediatric patients with SAA in Brazil. This was a retrospective study, which included 106 patients ≤18 years old who received a first BMT for SAA. All patients received bone marrow as graft source from an MRD (n = 69) or a URD (n = 37). Conditioning regimen was non‐myeloablative in 73.6% of cases, and GVHD prophylaxis comprised a calcineurin inhibitor plus methotrexate in 89.6% of patients. After a median follow‐up of 4.5 years after BMT, 81 patients are alive, with a 4‐year OS of 77% and no statistically significant difference between the MRD and URD groups (82% vs. 69%, respectively; P = .08). Grade III‐IV aGVHD at 6 months and cGVHD at 2 years were observed in 8% and 14% of cases, respectively, and were not statistically different between the groups. Twenty‐five (23%) patients died at a median of 2.9 months after BMT. Our study showed that 4‐year OS after BMT was not statistically different between MRD and URD recipients. This study shows that the outcomes of pediatric patients transplanted for SAA with a URD in Brazil are approaching those of MRD transplants. In contrast, OS after MRD BMT was lower than we would expect based on previous reports. The wide range of preparatory regimens used by the study centers highlights the need for standardized protocols for these children. Our findings provide a benchmark for future studies focused on improving BMT outcomes in this setting in Brazil.

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Allogeneic hematopoietic cell transplantation for severe aplastic anemia: similar long-term overall survival after transplantation with related donors compared to unrelated donors

Cited by 15 publications

References 9 publications

Effect of Stem Cell Source and Dose on Allogeneic Hematopoietic Stem Cell Transplantation in Adult Patients with Idiopathic Aplastic Anemia: Data from the Korean Aplastic Anemia Trials

Effect of Stem Cell Source and Dose on Allogeneic Hematopoietic Stem Cell Transplantation in Adult Patients with Idiopathic Aplastic Anemia: Data from the Korean Aplastic Anemia Trials

Allo-HSCT compared with immunosuppressive therapy for acquired aplastic anemia: a system review and meta-analysis

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