Lay Summary: Allogeneic stem cell transplantation is standard of care for younger patients with MDS.
Allogeneic stem cell transplantation is standard of care for younger patients with MDS. Age 60 is considered appropriate for it. The guidelines recommend that all patients < 65 years should be assessed for fitness/eligibility for allogeneic SCT as soon as possible after diagnosis, as SCT outcome is improved if performed early. If eligible and a sibling donor is available, it is recommended that patients < 50 years are offered ablative allogeneic SCT (evidence grade B, level IIb) and patients > 50 < 65 years are considered for non-ablative allogeneic SCT, within clinical trials where available (evidence grade C, level IV). The kind of transplant had not been defined, but the filed has moved toward older ages for both of these types of transplant.
Until the mid 1990s few patients over 55 years were offered allogeneic HSCT63 due to higher NRM in older patients. As only about 25% of MDS patients are younger than 60 years, efforts have focused on reducing the elevated risk of NRM associated with older age by modifying conditioning regimens (as discussed above), supportive care and complication management. Recent successfully transplanted cohorts included patients with median ages of 55–60 years, with some patients older than 70 years.
Patients with no sibling donor, but with an unrelated donor should also be considered for ablative unrelated-donor SCT. Case series and retrospective analyses demonstrate similar disease-free and overall survival rates with myeloablative and non-myeloablative/reduced-intensity conditioning regimens. Two-, three-, and four-year overall survival rates are 33% versus 35%, 39% versus 33%, and 36% versus 27%, respectively, for individuals undergoing allogeneic HSCT with myeloablative or non-myeloablative/reduced-intensity therapy
NCCN 2015 on p.MD S-11 footnotes the high dose therapy recommendation with “Hematopoietic stem cell transplant (HSCT): Allogeneic-matched sibling including standard and reduced-intensity preparative approaches or MUD”.
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