Hematopoietic stem cell transplantation in children with hemoglobinopathies: A single-center experience

INTRODUCTION: Hematopoietic stem cell transplantation (HSCT) is a curative treatment for children with serious hematological disorders including thalassemia major (TM) and sickle cell disease (SCD). This study aimed to determine the frequencies of transplant-related morbidity and outcome in our tertiary health-care facility. MATERIALS AND METHODS: We retrospectively analyzed the clinical, laboratory, and outcome data of 32 cases of hemoglobinopathies (TM = 19 and SCD = 13) who underwent bone marrow (BM) transplantation between 2005 and 2016. SPSS was used for statistical analysis, and Kaplan–Meier curve was constructed for overall survival (OS) and event-free survival. RESULTS: The median age of the study population at the time of transplant was 7.8 (range 2–14 years). Male-to-female ratio was 1:1. All patients were received allogeneic full matched related donor transplantation. Conditioning regimen was Bu/Cy/antithymocyte globulin (ATG) for SCD and Flu/Bu/Cy/ATG for TM. Source of stem cell in all patients was bone marrow and median stem cell dose CD 34+ was 5.26 (range 2.9–9.9) × 106/kg. All patients were received cyclosporin and methotrexate for graft versus host disease (GVHD) prophylaxis. Acute and chronic GVHD were observed in 10 (31.25%) and 2 (6.3%) cases, respectively. The median time to absolute neutrophil count (ANC) engraftment was 22 days (range: 12–27), and the median time to platelet engraftment was 32 days (range: 18–45) in 28 patients and the remaining four patients had delayed platelet engraftment (62–90 days). Cytomegalovirus (CMV) reactivation was observed in 18 (56.3%) cases; majority were asymptomatic (n = 17/18; 94.4%). Three-year event-free survival (EFS) and OS were 90.6% and 93.7%, respectively, with median duration of follow-up 33.72 (± 25.3) months. CONCLUSION: The results of the pediatric HSCT for hemoglobinopathies at our institution are excellent and are comparable to published reports from international collaborative groups

Survival Outcome of Empirical Antifungal Therapy and the Value of Early Initiation: A Review of the Last Decade

Aim: This rapid systematic review aimed to collect the evidence published over the last decade on the effect of empirical antifungal therapy and its early initiation on survival rates. Methods: A systematic search was conducted in PubMed, Cochrane, Medline, Scopus, and Embase, in addition to a hand search and experts’ suggestions. Results: Fourteen cohort studies and two randomized clinical trials reporting the survival outcome of empirical antifungal therapy were included in this review. Two studies reported the association between early empirical antifungal therapy (EAFT) and survival rates in a hematological cancer setting, and fourteen studies reported the outcome in patients in intensive care units (ICU). Six studies reported that appropriate EAFT decreases hospital mortality significantly; ten studies could not demonstrate a statistically significant association with mortality rates. Discussion: The inconsistency of the results in the literature can be attributed to the studies’ small sample size and their heterogeneity. Many patients who may potentially benefit from such strategies were excluded from these studies. Conclusion: While EAFT is practiced in many settings, current evidence is conflicting, and high-quality studies are needed to demonstrate the true value of this approach. Meanwhile, insights from experts in the field can help guide clinicians to initiate EAFT when indicated

Clinical characteristics and treatment outcome of childhood acute lymphoblastic leukemia in Saudi Arabia: A multi-institutional retrospective national collaborative study

Background: Treatment of childhood acute lymphoblastic leukemia (ALL) has been available in Saudi Arabia (SA) for over 30 years; however, only limited data have been published from there. This study was conducted to establish processes for collaborative data collection and provide clinical characteristics and outcome of children with ALL in SA.Procedure: Clinical data for patients diagnosed from 2004 to 2008 were retrospectively collected at eight institutions and entered remotely into a custom-built database. Statistics regarding clinical and genetic characteristics and treatment outcome were calculated.Results: The 594 evaluable patients had a median age of 4.37 years and 56.4% were boys. Majority of patients had B-precursor ALL while 10.7% had T-ALL. CNS leukemia was present in 5.2% of patients. The distribution of common genetic abnormalities was similar to that reported from western populations, with 24.6% hyperdiploidy, 21% RUNX1-ETV6 positivity, 4.2% BCR-ABL1 positivity, and 2.5% with MLL gene rearrangement. Patients received risk-adapted therapy according to various protocols, although treatment strategies for the majority were similar. Five-year OS, RFS and EFS were 86.9%, 79.1%, and 73.3%, respectively. The OS for patients with pre-B ALL was significantly higher than for T-ALL (88.0% vs. 71.8%; P = 0.019, Log-Rank test). Patients with pre-B ALL categorized as low-risk by NCI/Rome criteria and those with hyperdiploidy had OS of 93.4% and 95.8%, respectively.Conclusions: The characteristics of childhood ALL in SA are similar to those observed in developed countries. Future prospective studies utilizing unified national protocols are needed to further improve the outcome of our patients