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Hematopoietic stem cell transplantation (HSCT) can be a successful treatment for patients with high-risk myelodysplastic syndrome (HR-MDS) or acute myeloid leukemia (AML).1 However, factors such as age, performance status, comorbidities, and patient frailty may influence transplant referral patterns and outcomes in this population. Currently, the proportion of eligible patients being referred for transplantation is not known in clinical settings.1
The MDS Hub has previously covered the American Society for Transplantation and Cellular Therapy recommendations for transplantation in patients with MDS. Here we present a summary of the findings from a prospective cohort study on the patterns of consideration for HSCT in patients with HR-MDS and AML.1 In addition, we also summarize a retrospective study of survival benefits with allogeneic-HSCT (allo-HSCT) versus decitabine, a hypomethylating agent (HMA) agent presented by Runzhi at the European Hematology Association (EHA) 2023.2
This analysis comprised data from the Connect Myeloid Disease Registry (NCT01688011), a multicenter, prospective, observational cohort study in patients with newly diagnosed MDS or AML.1 Cohorts included patients enrolled at community (CO), government (GOV), or academic (AC) sites. Eligible patients were:1
Patients were assessed for HSCT eligibility at baseline and at each follow-up visit. HSCT eligibility criteria included considered potentially eligible, not considered potentially eligible, or not assessed.
Overall, 778 patients were included, of which 66.3% were from the CO/GOV cohort and 33.7% from the AC cohort.1 Patients in the AC cohort were younger (p < 0.0001), more likely to reside in the Northeastern United States (p < 0.0001), and more likely to have a private insurance (p = 0.017) compared with the CO/GOV cohort (Table 1).
Table 1. Baseline characteristics*
Characteristics, % (unless otherwise specified) |
CO/GOV cohort (n = 516) |
AC cohort (n = 262) |
---|---|---|
Median age, (range) years |
73 (19–97) |
69 (34–90) |
Sex, male |
64.1 |
62.6 |
Not specified |
8.7 |
5.7 |
Primary insurance† |
||
Medicare |
66.9 |
57.0 |
Private |
24.7 |
32.8 |
Medicaid |
1.6 |
3.5 |
Other |
6.9 |
6.6 |
Overall comorbidity grade |
||
0 or 1 |
50.8 |
59.0 |
2 or 3 |
49.2 |
41.0 |
Diagnosis† |
|
|
HR-MDS |
50.4 |
30.9 |
IR-AML |
30.2 |
36.6 |
AR-AML |
19.4 |
32.4 |
AC, academic; AML, acute myeloid leukemia; AR, adverse risk; CO/GOV, community/government; HR, higher-risk; IR, immediate risk; MDS, myelodysplastic syndromes. |
Figure 1. Reasons for transplant ineligibility in CO/GOV versus AC cohort*
AC, academic; CO/GOV, community/government; ECOG PS, Eastern Cooperative Oncology Group performance status.
†≥1 is possible.
*Adapted from Tomlinson et al.1
This was a retrospective study comparing responses to allo-HSCT with decitabine in patients with MDS with increased blasts type I (MDS-IBI). Outcomes included overall survival (OS), event-free survival (EFS), non-relapse mortality (NRM), and cumulative rate of relapse or progression (CIR) at 1 year.
Figure 2. Survival rates in patients with MDS-IBI treated with allogeneic-HSCT vs decitabine*
Allo-HSCT, allogeneic stem cell transplant; MDS-IBI, myelodysplastic syndrome with increased blasts type 1.
*Adapted from Runzhi.1
The study by Tomlinson1 showed that many patients who may be potential candidates for allo-HSCT are either not being assessed or considered for transplantation in clinical settings. This suggests the need for increasing resources for transplantation to ensure all patients are appropriately screened and referred for allo-HSCT if appropriate. The study by Runzhi2 demonstrates significantly prolonged survival in patients with MDS-IBI who underwent allogeneic-HSCT compared with those administered decitabine.
Together, both studies provide an insight into the success of allo-HSCT and how factors influencing eligibility should be considered in all patients, and patients should not be disregarded ahead of assessment for HSCT due to age and comorbidities.1,2
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