Allogeneic hematopoietic stem cell transplantation for myelodysplastic syndrome in adolescent and young adult patients

Shimomura, Yoshimitsu; Hara, Masahiko; Konuma, Takaaki; Itonaga, Hidehiro; Doki, Noriko; Ozawa, Yukiyasu; Eto, Tetsuya; Uchida, Naoyuki; Aoki, Jun; Kato, Jun; Onishi, Yasushi; Takahashi, Satoshi; Fukushima, Kentaro; Nakamae, Hirohisa; Kawakita, Toshiro; Tanaka, Junji; Fukuda, Takahiro; Atsuta, Yoshiko; Ishikawa, Takayuki; Ishiyama, Ken

doi:10.1038/s41409-021-01324-8

Cited by 14 publications

(9 citation statements)

References 37 publications

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“…Several studies have investigated the association between conditioning intensity and transplant outcomes in patients with MDS. 9,[21][22][23][24][25] These studies have shown that RIC is associated with a lower NRM but a higher relapse rate than MAC, resulting in comparable survival. A prospective randomized control trial from the European Society for Blood and Marrow Transplantation (EBMT) compared RIC and MAC for 129 patients with MDS and revealed that RIC had equivalent transplant outcomes, including NRM, relapse rate, PFS and OS compared to MAC.…”

Section: Discussionmentioning

confidence: 98%

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The prognosis and risk factors for patients with complex karyotype myelodysplastic syndrome undergoing allogeneic haematopoietic stem cell transplantation

Shimomura,

Komukai,

Kitamura

et al. 2023

Br J Haematol

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SummaryAllogeneic haematopoietic stem cell transplantation (HCT) is the curative treatment for myelodysplastic syndrome with a complex karyotype (CK‐MDS). However, only a few studies have been limited to patients with CK‐MDS undergoing allogeneic HCT. This study aimed to identify the risk factors for patients with CK‐MDS undergoing allogeneic HCT. We included 691 patients with CK‐MDS who received their first allogeneic HCT. The overall survival (OS) was the primary end‐point, estimated using the Kaplan–Meier method. Prognostic factors were identified using a Cox proportional hazards model. The 3‐year OS was 29.8% (95% confidence interval [CI]: 26.3–33.3). In the multivariable analysis, older age (hazard ratio [HR]: 1.44, 95% CI: 1.11–1.88), male sex (HR: 1.38, 95% CI: 1.11–1.71), poor haematopoietic cell transplant comorbidity index (HR: 1.47, 95% CI: 1.20–1.81), red blood cell transfusion requirement (HR: 1.58, 95% CI: 1.13–2.20), platelet transfusion requirement (HR: 1.85, 95% CI: 1.46–2.35), not‐complete remission (HR: 1.55, 95% CI: 1.16–2.06), a high number of karyotype abnormality (HR: 1.63, 95% CI: 1.18–2.25) and monosomal karyotype (HR: 1.49, 95% CI: 1.05–2.12) were significantly associated with OS. Thus, the 3‐year OS of allogeneic HCT was 29.8% in patients with CK‐MDS, and we identified risk factors associated with poor OS.

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Section: Discussionmentioning

confidence: 98%

“…23,24 A retrospective study from the Center for International Blood and Marrow Transplantation Research conducted a comparison Univariate and multivariable analyses for overall survival.…”

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confidence: 99%

The prognosis and risk factors for patients with complex karyotype myelodysplastic syndrome undergoing allogeneic haematopoietic stem cell transplantation

Shimomura,

Komukai,

Kitamura

et al. 2023

Br J Haematol

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“…A Japanese retrospective study reported the outcomes of allogeneic HSCT in 645 patients with AYA and indicated that the 3-year OS and CIR after transplantation were 71.2% and 11.2%, respectively 36 . The OS following allogeneic HSCT in patients in the lower-risk group in our study was similar to that in the Japanese cohort, although Shimomura et al did not evaluate the mutation profiles.…”

Section: Discussionmentioning

confidence: 99%

Impact of gene alterations on clinical outcome in young adults with myelodysplastic syndromes

Konishi

Sadato

Toya

et al. 2023

Sci Rep

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Young adults with myelodysplastic syndrome (MDS) are rare, and the clinical significance of driver mutations has not yet been analysed. We analysed the gene mutations and copy number alterations (CNAs) in younger MDS patients using next-generation sequencing, targeting 68 genes that were recurrently mutated in myeloid malignancies, to investigate the correlation between their genetic alterations and clinical outcomes. We enrolled 55 patients retrospectively (aged < 50 years). At least one mutation was detected in 56% of the patients. The most frequently mutated genes were ASXL1 and RUNX1, 13% each. We defined higher-risk patients as those with ≥ 2 mutations, except for SF3B1 mutation, and/or CNA. The 3-year overall survival (OS) in patients with a higher-risk was lower than that in those with a lower-risk (50.8% vs. 71.8%, P = 0.024). Among the 44 transplant recipients, patients with higher-risk had a significantly lower OS and tended to have a higher cumulative incidence of relapse (CIR) than those with a lower-risk (3-year OS: 38.0% vs. 64.4%, P = 0.039; 3-year CIR: 44.0% vs. 24.1%, P = 0.076). Our results showed that genetic aberrations can predict clinical outcomes in younger MDS patients, despite the low rate of genetic mutations.

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“…1 Although allo-HSCT has progressed significantly in recent years, relapse after transplantation remains an important concern. 2 Previous studies showed that the cumulative incidence of relapse in patients with advanced MDS (refractory anemia and excess blasts in transformation and secondary acute myeloid leukemia) within 3 years was 23% to 43%, and multivariate analyses revealed that the percentage of blasts was a significant predictor of relapse. 3,4 Limited therapeutic options are available for cases of relapse after allo-HSCT.…”

Section: Introductionmentioning

confidence: 99%

“…Allogeneic hematopoietic stem cell transplantation (allo‐HSCT) is the only potentially curative approach that results in long‐term survival for patients with myelodysplastic syndrome with excess blasts (MDS‐EB) 1 . Although allo‐HSCT has progressed significantly in recent years, relapse after transplantation remains an important concern 2 . Previous studies showed that the cumulative incidence of relapse in patients with advanced MDS (refractory anemia and excess blasts in transformation and secondary acute myeloid leukemia) within 3 years was 23% to 43%, and multivariate analyses revealed that the percentage of blasts was a significant predictor of relapse 3,4 .…”

Section: Introductionmentioning

confidence: 99%

Poor pretransplantation minimal residual disease clearance as an independent prognostic risk factor for survival in myelodysplastic syndrome with excess blasts: A multicenter, retrospective cohort study

Wei

et al. 2023

Cancer

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Background Minimal residual disease (MRD) is an important prognostic factor for survival in adults with acute leukemia. The role of pretransplantation MRD status in myelodysplastic syndrome with excess blasts (MDS‐EB) is unknown. This study retrospectively analyzed the relationship between pretransplantation MRD status and long‐term survival. Materials and Methods Patients with MDS‐EB who underwent allogeneic hematopoietic stem cell transplantation (allo‐HSCT) from March 5, 2005, to November 8, 2020, were included. The relationship between pretransplantation MRD status and long‐term survival was analyzed using univariate and multivariate logistic regression models. Results Of 220 patients with MDS‐EB who underwent allo‐HSCT, 198 were eligible for inclusion in this multicenter, retrospective cohort study. Complete remission was attained in 121 (61.1%) patients, and 103 patients underwent detection of MRD pretransplantation, with 67 patients being MRD‐positive and 36 patients being MRD‐negative. The median follow‐up time was 16 months, the median age was 41 years (6–65 years), and 58% of the patients were men. The 3‐year disease‐free survival (DFS) and overall survival (OS) probabilities for all patients were 70.1% and 72.9%, respectively. For patients in complete remission, the 3‐year DFS and OS probabilities were 72.2% and 74.8%, respectively. Further analysis found that the 3‐year DFS rates of MRD‐negative and MRD‐positive patients were 85.6% and 66.5% (p = .045), respectively, whereas the 3‐year OS rates were 91.3% and 66.4% (p = .035), respectively. Univariate and multivariate analyses showed that poor pretransplantation MRD clearance was an independent prognostic risk factor for DFS and OS. Conclusion Poor pretransplantation MRD clearance is an independent prognostic risk factor for long‐term survival after allo‐HSCT for patients with MDS‐EB. Plain language summary Poor minimal residual disease clearance pretransplanation is an independent prognostic risk factor for long‐term survival after allogeneic hematopoietic stem cell transplantation for patients with myelodysplastic syndrome with excess blasts.

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Allogeneic hematopoietic stem cell transplantation for myelodysplastic syndrome in adolescent and young adult patients

Cited by 14 publications

References 37 publications

The prognosis and risk factors for patients with complex karyotype myelodysplastic syndrome undergoing allogeneic haematopoietic stem cell transplantation

The prognosis and risk factors for patients with complex karyotype myelodysplastic syndrome undergoing allogeneic haematopoietic stem cell transplantation

Impact of gene alterations on clinical outcome in young adults with myelodysplastic syndromes

Poor pretransplantation minimal residual disease clearance as an independent prognostic risk factor for survival in myelodysplastic syndrome with excess blasts: A multicenter, retrospective cohort study

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