Abstract
Myelodysplastic syndromes are clonal disorders with morphological dysplasia, a variable degree of cytopenia and a risk of transformation to acute myeloid leukemia. Prognosis is very variable and is defined by blast count, cytopenia, cytogenetics and more recently by somatic mutations, with IPSS or revised IPSS score being the most widely used to assess disease risk. HSCT remains the only curative treatment to date, with high-risk patients obtaining the biggest benefit. However, NRM should be carefully assessed before indicating the transplant in this usually old population, where organ toxicity and comorbid conditions are to be considered. Multi-domain assessment tools, such as CGA (comprehensive geriatric assessment) and EBMT score, are useful in this context and might guide physician decisions regarding the transplant. Indeed, with the development of reduced intensity conditioning regimens, the number of patient candidates for an HSCT has increased. Regarding pre-transplant treatment, patients with a blast excess > 10% might be treated with HMAs or chemotherapy, although there are no randomized trials confirming the benefit of this approach, even when achieving a complete response. Concerning donor choice, matched sibling donors continue to be the first option, although matched unrelated donors, and more recently haploidentical donors, have proven to be valid options and should be offered in the absence of a related donor. Relapse remains the main cause of transplantation failure. MRD assessment and pre-emptive or prophylactic use of HMA or other targeted inhibitors with or without DLI are accepted strategies to reduce relapse risk, but the prognosis in this context remains dismal, and is the subject for several ongoing clinical protocols.
Highlights
Myelodysplastic syndromes (MDS) are characterized by dysplastic morphology of hematopoietic cells associated with clonal hematopoiesis, peripheral blood cytopenia, and a propensity to progress to acute myeloid leukemia
Regarding the donor choice between matched sibling donor (MSib) and unrelated (MUD), a CIBMT registry study reported that treatment failure is similar among MDS patients who received a transplant from MSib as compared to MUD [43]
Hematopoietic stem cell transplantation (HSCT) is a curative therapy for MDS, but non-relapse mortality (NRM) remains a major obstacle to the success of this treatment
Summary
Myelodysplastic syndromes (MDS) are characterized by dysplastic morphology of hematopoietic cells associated with clonal hematopoiesis, peripheral blood cytopenia, and a propensity to progress to acute myeloid leukemia. Transformation into acute leukemia is the main cause of death in patients with higher risk. Hematopoietic stem cell transplantation (HSCT) remains, to date, the only potentially curative therapy, albeit with a treatment-related mortality between 15 and 50%, according to specific risk factors [6,7,8]. Some low-risk patients with persistent disease might live for years with supportive therapy and a relatively good quality of life. Hypomethylating agents (HMAs) have been reported to increase median survival and decrease transformation into leukemia in higher risk patients, and should be balanced with the potential effect of transplantation [9]. Hemato 2021, 2 marrow blast percentage is a risk factor for disease progression, but there is no randomized trial demonstrating a benefit for pre-transplant cytoreduction. This review will detail the current evidence for transplant strategies in MDS patients
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