Abstract
Allogeneic hematopoietic stem cell transplantation is the sole curative therapy for myelodysplastic syndrome (MDS). However, there is concern regarding graft failure and relapse in patients who undergo cord blood transplantation (CBT). We conducted a retrospective study of the CBT outcomes in MDS patients using the Japanese Data Center for Hematopoietic Cell Transplantation database. Seven hundred fifty-two de novo MDS patients of ≥18 years of age (median, 58 years) undergoing their first CBT between 2001 and 2015 were examined. Two-thirds of the patients were male, and were RAEB. The cumulative incidences of neutrophil and platelet engraftment at day 100 were 77 and 59%, respectively. The 3-year overall survival (OS) was 41% and the median survival of the patients was 1.25 years. A multivariate analysis of pre-transplant variables showed that the age, gender, cytogenetic subgroups, number of RBC transfusions, HCT-CI and year of CBT significantly influenced the outcome. The cumulative incidence of acute graft-versus-host disease (aGVHD) and chronic GVHD (cGVHD) was 32 and 21%, respectively. A survival benefit was observed in patients who developed cGVHD, but not aGVHD. Our results suggest that CBT is an acceptable alternative graft and that a graft-versus-MDS effect can be expected, especially in patients who develop cGVHD.
Highlights
Over the long term, there are no effective treatment for the patients with myelodysplastic syndrome (MDS)
The use of cytotoxic agents can be considered for MDS subtypes with increased blasts; even if complete remission is obtained by combination chemotherapy which is used for the treatment of acute leukemia, the status does not last long, and subsequent event-free survival was not good[2,3]
The following factors were included in the analysis: age at cord blood transplantation (CBT), gender, MDS subtype, cytogenetic subgroup, International Prognostic Scoring System (IPSS) classification, performance status (PS), blood type, serological results for HLA-A/B/DRB1, number of RBC and platelet transfusions prior to CBT, type of bridging therapy between the diagnosis and the CBT, effect of bridging therapy, positivity for anti-HLA antibody, hematopoietic cell transplantation-specific comorbidity index (HCT-CI), conditioning regimen, date of CBT, prophylactic agent for graft-versus-host disease (GVHD), date and severity of the development of acute and chronic GVHD, date of relapse, date of last follow-up and survival
Summary
There are no effective treatment for the patients with myelodysplastic syndrome (MDS). The outcome of supportive care for higher-risk MDS cases is poor; the prognosis of patients with intermediate-2 and high classifications according to the International Prognostic Scoring System (IPSS) is 1.2 years and 0.4 years, respectively[1]. The use of cytotoxic agents can be considered for MDS subtypes with increased blasts; even if complete remission is obtained by combination chemotherapy which is used for the treatment of acute leukemia, the status does not last long, and subsequent event-free survival was not good[2,3]. Even though the launch of new drugs such as hypomethylating agents and multikinase inhibitors has improved the overall survival of MDS patients in recent years, it would be difficult to obtain a cure with these agents[4,5]. Most hematologists recognise that allogeneic hematopoietic stem cell transplantation (allo-SCT) is the sole curative therapy.
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