Transplant Referral Patterns for Patients (Pts) with Newly Diagnosed (ND) Higher-Risk (HR) Myelodysplastic Syndromes (MDS), and European Leukemianet (ELN) 2010 Intermediate-Risk (IR) or Adverse-Risk (AR) Acute Myeloid Leukemia (AML) in the Connect® MDS/AML Registry

Abstract

Hematopoietic stem cell transplantation (HSCT) is often indicated and formal transplant referral guidelines exist for pts with AML and HR MDS. However, the proportion of transplant-eligible pts not referred is largely unknown. We therefore assessed HSCT referral patterns, including potential barriers to referral, in pts with ND MDS and AML enrolled in the Connect® MDS/AML Disease Registry (NCT01688011), a large, US, multicenter, prospective observational cohort study of pts with ND AML (aged ≥ 55 years) or MDS (aged ≥ 18 years). We evaluated pt data, including transplant referral decisions collected at enrollment and every 3 months, from pts enrolled from Dec 2013 to Jul 2019. HR MDS and AML pts were included; AML pts were grouped by ELN 2010 risk groups into IR AML (Int-1 and Int-2 risk), or AR AML (adverse risk). Differences in transplant referral rates were assessed using a chi-square t-test (significance <i>P</i> < 0.05) in pts with HR MDS. Baseline characteristics of 720 pts with IR AML, AR AML, and HR MDS enrolled at 22 academic (AC) and 119 community/government (CO/GOV) sites are shown (Table). Median time in the Registry was 8.1 (range 0.1–62.9) months. Pts at CO/GOV sites were less likely to be considered for transplant by the treating physician than pts at AC sites (28% vs 43%; <i>P</i> < 0.001); of those considered, there was no difference in the transplant rates between the 2 site types (43% vs 36%; <i>P</i> = 0.290). For 358 pts deemed transplant ineligible, the most common reasons were comorbidities at AC sites and age at CO/GOV sites (Fig. A). Median age of ineligible pts was 77 (range 56–94) years. 7 pts were ineligible because of favorable cytogenetics; however, all had HR MDS, IR AML, or AR AML. In a subset of 262 pts (AC = 79; CO/GOV = 183) identified by investigators as likely potential transplant candidates based on age, comorbidity grade, frailty score, and ECOG PS, pts at AC sites were more likely to be considered for transplant than pts at CO/GOV sites (58% vs 43%; <i>P</i> = 0.020) with age the most common reason for transplant ineligibility (Fig. B) at both sites. Rates of consideration for transplant were lower at CO/GOV sites than AC sites. Among pts identified as potential transplant candidates, rates of consideration were lower at CO/GOV sites suggesting that further education may be needed to ensure referral of all pts who would benefit from transplant.