A new population-based study suggests that among patients with myelodysplastic syndromes (MDS), many who are candidates for allogeneic hematopoietic stem cell transplantation (HCT) do not reach HCT, for a variety of reasons. The study results were published in the journal Bone Marrow Transplantation.
The study involved patients who were included in the National Swedish quality registry (cohort 1), in addition to patients treated at the Karolinska University Hospital in Stockholm, Sweden (cohort 2). All included patients were transplant candidates who had MDS or MDS/MPNs. In addition to other statistical tests, multivariate analysis was used to evaluate features that may be linked to failure to reach HCT.
Cohort 1 had 289 evaluable patients, and cohort 2 had 130 evaluable patients. The median age at diagnosis was 58 years for cohort 1, and the median age during the decision to undergo HCT was 60 years for cohort 2. The Revised International Prognostic Scoring System (IPSS-R) risk group was intermediate, high, or very high for most patients in both cohorts. The IPSS-R prognostic subgroup by karyotype was considered poor in 15% of patients in cohort 1 and 14% of patients in cohort 2.
Overall, 25% of patients in cohort 1, and 22% of patients in cohort 2, failed to reach HCT. For cohort 2, causes of failure were available, which included progressive or refractory disease in nearly half (47%) of the patients who did not reach HCT, a lack of a suitable donor in 21% of cases, identification of a comorbidity in 18% of cases, and infection in 14%.
Multivariate analysis revealed several factors that appeared related to failure to reach HCT. In cohort 1, the researchers considered included a marrow blast count of 0% to 4.9% (odds ratio [OR], 2.59; P =.003), age at diagnosis (OR, 1.05; P =.003), and very poor cytogenetics (OR, 2.0; P =.05) to be independent risk factors for failure to reach HCT. In cohort 2, age at diagnosis was the only independent risk factor identified for failure to reach HCT (OR, 1.05; P =.04). Additionally, in cohort 1, a hemoglobin level of ≥10 g/dL was linked to a lower risk of failing to reach HCT (OR, 0.45; P =.02).
In cohort 1, receipt of HCT was associated with longer overall survival (OS) when measured at a median follow-up of 29 months from diagnosis. In this cohort, the median OS for patients who had HCT was 83 months, compared with 14 months for patients who did not reach HCT (P <.001). Survival benefits with HCT were associated with intermediate, high, or very high IPSS-R risk status in this cohort. In cohort 2, with a median follow-up of 41.5 months after the decision to undergo HCT, the median OS with HCT was 105 months with HCT and 26 months without HCT (P <.001).
“In summary, we have in a population-based cohort shown that there is a significant proportion of patients failing to reach HCT underlining the difficulties to bring patients to HCT,” the researchers concluded in their report.
Lindholm C, Olofsson E, Creignou M, et al. Failure to reach hematopoietic allogenic stem cell transplantation in patients with myelodysplastic syndromes planned for transplantation: a population-based study. Bone Marrow Transplant. Published online February 2, 2022. doi:10.1038/s41409-022-01582-0