Saudi Arabia is the largest of the Arabian Gulf countries with a total population of 33.41 million as of 2017. This report summarizes the experience from four leading tertiary care hematopoietic stem cell transplantation (HSCT) centers in Saudi Arabia representing more than 90% of all HSCTs performed in the country. Between 1984 and 2016, a total of 6,184 HSCTs were performed. Of these, 3,586 HSCTs were performed in adults and 2,598 HSCTs were performed in pediatric patients. Malignancy was the main indication for transplantation (47%). While most transplants were performed from an identical sibling donor, HSCTs from cord blood, unrelated and, more recently, haploidentical donors have also been performed. Relative shortage of HSCT bed capacity is perceived to be a limiting factor in Saudi Arabia. Lately, more HSCT centers are emerging with rapid growth, which may significantly improve the access to HSCT in the country in the near future.
Objective: Pediatric acute myeloid leukemia (AML) with KMT2A rearrangement is seen in 15-20% of patients. KMT2A has been shown to rearrange with more than 80 distinct partner genes. In this study we examined the pattern of the various KMT2A rearrangements and their treatment outcomes in our patient population. Methods: We retrospectively analyzed pediatric AML patients with KMT2A rearrangement seen in our institution between January 2005 and December 2015. Results: There were 18 evaluable patients with equal genders. The median age was 4.12 years (range 0-13.5). FAB classification M5 was the most common morphology. Common translocation partner was KMT2A/MLLT3. Ten patients were treated with chemotherapy only and 8 with hematopoietic stem cell transplantation (HSCT). There were 50% patients alive in each group. Conclusion: KMT2A patients can be treated with both chemotherapy and HSCT. The different fusion partners can lead to heterogeneous outcomes in children with KMT2A rearranged AML. Further prospective studies are needed to delineate the high-risk sub-sets in KMT2A rearranged AML that will benefit from HSCT.
Key Points A 6-year overall survival of 61% was observed in leukodystrophy patients after cord blood transplantation. Mismatched cord blood donors, symptomatic disease, and lower PS before cord blood transplantation were predictors of lower survival.