While exagamglogene autotemcel (Casgevy) and lovotibeglogene autotemcel (Lyfgenia) have been approved by the US Food and Drug Administration (FDA) as the first cell-based gene therapies for the treatment of patients 12 years of age and older with sickle cell disease (SCD), this treatment is not universally accessible. Allogeneic hematopoietic stem cell transplant (HSCT) has the potential to eradicate the symptoms of patients with SCD, but a significant obstacle in HSCT for SCD is the availability of suitable donors, particularly human leukocyte antigen (HLA)-matched related donors. Furthermore, individuals with SCD face an elevated risk of complications during stem cell transplantation due to SCD-related tissue damage, endothelial activation, and inflammation. Therefore, it is imperative to consider optimal conditioning regimens and investigate HSCT from alternative donors. This review encompasses information on the use of HSCT in patients with SCD, including the indications for HSCT, conditioning regimens, alternative donors, and posttransplant outcomes.

Background: Gastroesophageal reflux disease (GERD) is one of the most common gastrointestinal pathology in infants and young children. Ultrasonography (US) has been considered to be a reliable diagnostic tool for GERD but the severity of GERD and the clinical implications based on imaging findings has not been evaluated. Aims: To compare the diagnostic value of lower esophageal US with that of barium swallow in demonstrating the severity of GERD. Materials and methods: Fifty one pediatric patients, age between 1 month to 12 years, 34 male and 17 female with clinical suspicion of GERD were included. The patients were initially submitted to barium swallow (BS) and subsequently to transabdominal US. During BS, the number of gastroesophageal reflux episodes was documented in a 5-minute period. Transabdominal US documented the number and duration of reflux episodes during a 5-minute period, the angle of His, mucosal thickness, and intraabdominal esophageal length (IAEL). Results: Duration and number of reflux episodes in US were significantly higher in patients that had severe gastroesophageal refluxes at BS. At US the cutoff point of 9.5 seconds (sensitivity 80%, specificity 60%) for reflux duration and more than 2 episodes in 5 minute ultrasound study (sensitivity 75%, specificity 58%) were defined to correlate with severe gastroesophageal reflux at BS.The angle of His, the esophageal wall mucosal thickness, and the IAEL did not cor- relate with the severity of GERD detected in BS. Conclusion: US can predict the severity of GERD. Therefore, except in the case of specific patients in whom mechanical causes are suspected to be responsible for GERD, BS can be replaced by US.

Male infertility in β-thalassemia patients is typically considered to be the consequence of iron deposition in the endocrine glands. Adult male patients with β-thalassemia, on regular blood transfusions, are prone to developing acquired hypogonadism. The aim of this study was to evaluate the fertility indicators in male patients with β-thalassemia major and intermedia. In this study we evaluated testicular volume, semen parameters and serum FSH, LH, and Testosterone concentrations in 62 male patients in reproductive age, with major and intermedia thalassemia, at a tertiary care hospital in Tehran, Iran. The range of serum ferritin level in our patients was from 182 to 11053 ng/mL (mean 2067 ng/mL). The mean concentration of sperm was 61.04 million per milliliter. The mean volume of right and left testes was 11.4 cc and 11.7 cc, respectively. Those patients who had lower testicular volumes significantly had lower sperm concentration, lower percents of motile and also lower percents of normal morphologic sperms (p = 0.04). The frequency of hypogonadism was significantly higher in patients whose testicular volume was lower (p = 0.02). Hypogonadism and hypothyroidism were seen in 22.6% and 17.7% of patients, respectively. Patients with hypogonadism had significantly lower ejaculate volume, lower sperm concentration, lower percents of motile and progressively motile sperms and also lower percents of normal morphologic sperms (p = 0.001). This study suggests that in thalassemic men, concentrations of serum testosterone, LH, FSH has significant correlation with sperm parameters and testicular volume.

Introduction: Recently, a relationship has been demonstrated between red blood cell distribution width (RDW) and mortality risk in critically ill patients although the exact mechanism of this association is still vague. However, the impact of changes in RDW on sepsis and its outcome in critically ill patients has not been widely studied. Therefore, we studied the prognostic impact of changes in RDW in critically ill pediatric patients with sepsis. Methods: A total of 304 patients who were admitted to pediatric intensive care unit were selected to participate in this study. The changes in RDW on the day of admission and 4 and 8 days after admission in PICU were documented and their relationship with SIRS positivity, sepsis, and mortality were analyzed. Results: The mortality rate in our patients was 10.5%. In total, 39.8% of patients were SIRS positive and 50.4% fulfilled the criteria of sepsis. The mean of RDW at the time of admission, on Day 4 and on Day 8 of admission was 14.8%, 16.1%, and 16.6%, respectively. At the time of admission, RDW had a significant correlation with mortality and SIRS positivity, but RDW measured on Days 4 and 8 of admission did not correlate with neither of them. Neither of RDW0, RDW4, nor RDW8 did correlate with sepsis criteria fulfillment. ΔRDW day 4-adm > 0.2%, ΔRDW day 8-adm > 0.2%, ΔRDW day 8-day 4 > 0.2% exhibited no correlation with SIRS positivity, sepsis, and mortality. Conclusions: We found that an increase in RDW from baseline during the first 4 and 8 days after admission of critically ill pediatric patients did not correlate with their mortality, SIRS positivity, and sepsis. However, elevated baseline RDW is a valuable prognostic marker in patients with sepsis. Keywords: Sepsis; RDW; Mortality; SIRS