Outcome and factors affecting survival of childhood myelodysplastic syndrome; single centre experience

Abstract

Introduction: Although many therapeutic options have been attempted for pediatric myelodysplastic syndrome (MDS), still hematopoietic stem cell transplant (HSCT) is the only curative therapy. We described the outcome of these patients and the factors affecting the overall survival (OS) and event free survival (EFS). Methods: This is a retrospective descriptive study included 44 patients with MDS who presented to Children Cancer Hospital Egypt (CCHE-57357), between July 2007 and December 2015. HSCT was offered to patients who had HLA matched family donors. Results: Eighteen patients (40.9%) had refractory cytopenia of childhood (RCC), 19 (43.2%) had refractory anemia with excess blasts (RAEB), and 7 (15.9%) had refractory anemia with excess blasts in transformation/myelodysplastic related acute myeloid leukemia (RAEB-T/MDR-AML). Cytogenetic abnormalities were observed in 54.8% of evaluable patients, in which monosomy 7 was the commonest abnormality observed (26.1%). Allogeneic HSCT from HLA matched donors was performed in 9/44 patients. The Average CD34 + count was 3.57 × 106/Kg recipient weight. The 2-year OS and EFS for the whole cohort were 43.3% and 33.8%, respectively, and for the transplanted patients were 75% and 60%, respectively. Univariate analysis showed improved 2 years-OS and EFS in patients who underwent HSCT as compared to those who didn't (75% versus 35.4% for OS, P = 0.088 and 60% versus 27.5%, for EFS, P = 0.074). Conclusion: Allografts from either matched related or unrelated or even haplo-identical donors should be offered for pediatric MDS patients, as HSCT is the only curative option for those patients. Further collaborative efforts, a more precise molecular diagnosis, as well as large-scale prospective therapeutic trials are required to develop standard diagnostic and therapeutic guidelines for such rare disease. Keywords: Myelodysplastic syndrome, MDS, Pediatric MDS, Hematopoietic stem cell transplantation, HSCT