Allogeneic Hematopoietic Cell Transplantation for Adult Chronic Myelomonocytic Leukemia

Abstract

Allogeneic hematopoietic cell transplantation (HCT) is potentially curative for patients with chronic myelomonocytic leukemia (CMML), however, few data exist regarding prognostic factors and transplant outcomes. We performed this retrospective study to identify prognostic factors for post-transplant outcomes. The CMML-specific prognostic scoring system (CPSS) has been validated in subjects receiving non-transplant therapy and was included in our study. From 2001–2012, there were 209 adult subjects who received HCT for CMML reported to the Center for International Blood and Marrow Transplant Research (CIBMTR). The median age at transplant was 57 years (range 23–74). Median follow up was 51 months (range, 3–122). On multivariate analyses, CPSS scores, Karnofsky performance status (KPS), and graft source were significant predictors of survival (p=0.004, p=0.01, p=0.01, respectively). Higher CPSS scores were not associated with disease-free survival, relapse, or transplant-related mortality. In a restricted analysis of subjects with relapse following HCT, those with intermediate-2/high risk had a nearly two-fold increased risk of death after relapse compared to those with low/intermediate-1 CPSS scores. Respective 1, 3 and 5-year survival rates for low/intermediate-1 risk subjects were 61% (95% confidence interval [CI], 52%–72%), 48% (95% CI, 37%–59%), and 44% (95% CI, 33%–55%), and for intermediate-2/high risk subjects were 38% (95% CI, 28%–49%), 32% (95% CI, 21% – 42%), and 19% (95% CI,8%–29%). We conclude that higher CPSS score at time of transplant, lower KPS, and a bone marrow (BM) graft are associated with inferior survival after HCT. Further investigation of CMML disease-related biology may provide insights into other risk factors predictive of post-transplant outcomes.

Stem cell transplantation for myeloproliferative diseases in the era of molecular therapy

“…Results in CMML patients treated with allo-HSCT depend on the pre-transplant risk scores (HCT-specifi c CPSS), as shown by IBMTR group in 209 patients [16]. I.e., adjusted disease-free survival, starting at the time of transplant, at a median follow-up of 51 months.…”

Advances in chronic myelomonocytic leukemia and future prospects: Lessons learned from precision genomics

“…A single‐institution study found advanced age, increased HCT comorbidity index and adverse‐risk cytogenetics to have an independent adverse prognostic impact with greater mortality and decreased relapse‐free survival . Retrospective data from Center for International Blood and Marrow Transplant Research validated the CPSS‐prognostic system in 209 CMML patients who were treated with HSCT, and found Karnofsky performance status, graft source and CPSS score to be independent predictors of survival in a multivariable model . Recently in MDS, mutation clearance detected in the bone marrows 30 days after allogeneic HSCT, correlated with a lower risk of disease progression .…”

Allogeneic hematopoietic stem cell transplantation for the treatment of BCR-ABL1-negative atypical chronic myeloid leukemia and chronic neutrophil leukemia: A retrospective nationwide study in Japan

“…Attempts to statistically evaluate prognostic impacts on post-transplant outcomes among patients with aCML were unsuccessful due to the small number of patients in the present study. We stratified OS after transplantation by the prognostic factors previously identified for aCML itself and MDS/MPN other than aCML [7,[41][42][43][44][45][46][47][48][49]: age at transplantation, the Karnofsky Performance Status (KPS), blast percentages in peripheral blood, white blood cell counts, hemoglobin levels, transfusion dependency, karyotype, the presence of splenomegaly, type of donor source, donor/recipient sex match, and the interval from the initial diagnosis to allo-HSCT. Regarding aCML, 1-year OS were 80.0% (95% CI, 20.4 to 96.9%) and 44.4% (95% CI, 13.6 to 71.9%) in patients using HLAmatched-related (n=5) and alternative donors (i.e.…”