Background: Hematopoietic stem cell transplantation (HCT) is indicated for patients with higher-risk (HR) myelodysplastic syndromes myelodysplastic syndromes: (my-eh-lo-diss-PLASS-tik SIN-dromez) A group of disorders where the bone marrow does not work well, and the bone marrow cells fail to make enough healthy blood cells. Myelo refers to the bone marrow. Dysplastic means abnormal growth or development. People with MDS have low blood cell count for at… (MDS) and acute myeloid leukemia acute myeloid leukemia: (uh-KYOOT my-uh-LOYD loo-KEE-mee-uh) A cancer of the blood cells. It happens when very young white blood cells (blasts) in the bone marrow fail to mature. The blast cells stay in the bone marrow and become to numerous. This slows production of red blood cells and platelets. Some cases of MDS become… (AML). Age, performance status, patient frailty, comorbidities, and non-clinical factors (eg, cost, distance to site) are all recognized as important clinical factors that can influence HCT referral patterns and patient outcomes. However, the proportion of eligible patients referred for HCT in routine clinical practice is largely unknown.
Objective: This study aimed to assess patterns of consideration for HCT among patients with HR-MDS and AML enrolled in the Connect® Myeloid Disease Registry, at community/government (CO/GOV)- or academic (AC)-based sites, as well as to identify factors associated with transplant referral rates.
Study design: We assessed patterns of consideration for, and completion of, HCT among patients with HR-MDS and AML enrolled between December 12, 2013 and March 6, 2020 in the Connect® Myeloid Disease Registry at 164 CO/GOV and AC sites. Registry sites recorded whether patients were considered for transplant at baseline and at each follow-up visit. The following answers were possible: "considered potentially eligible", "not considered potentially eligible", or "not assessed". Sites also recorded whether patients subsequently underwent HCT at each follow-up visit. Comparison of transplant consideration rates between CO/GOV and AC sites was performed using multivariable logistic regression analysis with covariates for age and comorbidity.
Results: Among the 778 patients with HR-MDS or AML enrolled in the Registry, patients at CO/GOV sites (27.9%) were less likely to be considered potentially eligible for HCT than patients at AC sites (43.9%; (P < .0001). Multivariable logistic regression analysis with factors for age (<65 versus ≥65 years) and ACE-27 comorbidity grade (<2 versus ≥2) demonstrated that patients at CO/GOV sites were significantly less likely to be considered potentially eligible for transplant than those at AC sites (odds ratio: 1.6, 95% confidence interval [CI], 1.1-2.4, P = .0155). Of patients considered eligible for transplant, 45.1% (65/144) and 35.7% (41/115) of patients at CO/GOV and AC sites, respectively, underwent transplantation (P = .12). Approximately half of all patients at CO/GOV (50.1%) and AC (45.4%) sites were not considered potentially eligible for HCT; the most common reasons were age at CO/GOV sites (71.5%) and comorbidities at AC sites (52.1%). Across all sites, 17.4% of patients across all sites were reported as not assessed (and thus not considered) for transplant by their treating physician (20.7% at CO/GOV and 10.7% at AC sites; P = .0005).
Conclusions: These findings suggest many patients with HR-MDS and AML who may be candidates for HCT are not receiving assessment or consideration for transplant in clinical practice. In addition, treatment at CO/GOV sites and age are still significant barriers to ensuring all potentially eligible patients are assessed for HCT.
Keywords: Acute myeloid leukemia; Bone marrow Bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. transplantation; Clinical practice; Hematologic malignancies: Myelodysplastic syndromes; Management and treatment.