The aim of this study is to describe the health status, health care received, and their impact on the quality of life in patients with hemophilia. Patients with severe factor VIII or IX deficiency without inhibitors or other chronic disease were enrolled. Turkish version of the Hemophilia-Specific Quality of Life Index (Haemo-QoL) questionnaire was administered to the pediatric patients aged 4 to 16 years and Haem-A-QoL to the adult patients. Joints were evaluated according to the World Federation of Hemophilia (WFH) orthopedic joint scores.Thirty-nine children/adolescents and 31 adult patients were enrolled. Mean Haemo-QoL scores were 39.6 +/- 15.0 for the children and mean Haem-A-QoL 47.4 +/- 14.1 for the adult patients, respectively. Internal consistency reliability was generally sufficient. Total Cronbach's alpha coefficient was >.70 (range .77-.96) in all the age groups. Mean total WFH orthopedic joint scores were 1.83 +/- 2.7, 4.9 +/- 4.96, and 6.94 +/- 6.15 in 4-7, 8-12, and 13-16-year-old groups, respectively. They were more impaired in the adult patients (16.23 +/-14.12). These results show that the Turkish version of the Haemo-QoL and Haem-A-QoL are reliable instruments to measure the quality of life in the pediatric and adult patients with severe hemophilia. When compared to the Haemo-QoL scores of an international multicenter West European study of children, quality of life in the Turkish patients were more impaired in the subscales of physical health, feeling, view, school and sport, and treatment as well as more impaired WFH joint scores. The authors recommend primary factor prophylaxis and encouraging the patients to learn home treatment to improve joint scores and quality of life.

Background Despite the increasing performance of allogeneic hematopoietic cell transplantation over the last decades, graft‐versus‐host disease (GVHD) remains the main cause of morbidity and mortality. The efficacy of ruxolitinib against GVHD has been demonstrated in adult studies; however, very few studies have been conducted in children. Procedure This study aimed to evaluate the efficacy of ruxolitinib in 29 children with steroid‐refractory acute or chronic GVHD. Twenty‐five (87%) patients received at least three different immune modulator agents, including methylprednisolone, before initiating ruxolitinib. Results All grade 2 acute GVHD patients completely responded to ruxolitinib treatment; 82% of high‐grade (3‐4) acute GVHD patients and 80% of chronic GVHD (moderate‐severe) patients had at least a partial response. Of seven patients with bronchiolitis obliterans, five had a partial response after ruxolitinib. Of 29 patients, 22 were administered steroids at any time in the first month of acute GVHD or the first three months of chronic GVHD during ruxolitinib usage, which was significantly tapered by the end of the observation period. Conclusion Steroid‐refractory acute and chronic pediatric GVHD patients treated with ruxolitinib had a high overall response rate, with the additional benefit of steroid sparing.

Neonatal thrombocytopenia is one of the most common hematologic disorders in neonatal intensive care units (NICUs). The purpose of this study was to determine the prevalence of thrombocytopenia and whether thrombocytopenia has an effect on the occurrence of intraventricular hemorrhage (IVH) ≥ grade 2 and on mortality rate. This study was carried out retrospectively in neonates admitted to NICU of Cumhuriyet University in Sivas, Turkey, between 2009 and 2012. Among 2218 neonates evaluated, 208 (9.4%) developed thrombocytopenia. The prevalence of IVH ≥ grade 2 was more in infants with thrombocytopenia (7.2%) than in those without thrombocytopenia (4.4%), although this was not statistically significant (P = .08). In univariate analysis, IVH ≥ grade 2 was higher in cases with very severe thrombocytopenia (35.7%, n = 5) than in those with mild (2.1%, n = 2), moderate (4.7%, n = 3), and severe thrombocytopenia (15.2%, n = 5) (P = .04). Multivariate logistic regression analysis showed that birth weight <1500 g (OR 6.2, 95% CI 3.4-9.8; P = .0001), gram-negative sepsis (OR 2.5, 95% CI 1.8-4.2; P = .01), very severe thrombocytopenia (OR 1.3, 95% CI 1.1-2.1; P = .03), and platelet transfusion ≥2 (OR 7.3, 95% CI 4.1-12.1; P = .001) were significant risk factors for mortality. The results of our study suggest that outcomes of neonates with thrombocytopenia depend not only on platelet count but also on decreased gestational age or birth weight, prenatal factors, and sepsis.

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Background Post‐Cy administration for GVHD prophylaxis in unmanipulated haploidentical HSCT has resulted in improved outcomes in recent years. Studies in children are lacking and accordingly we present the outcomes of 62 haploidentical transplantation for high‐risk children. Procedure We retrospectively assessed 62 transplants in 60 patients who underwent haploidentical‐related HSCT with unmanipulated stem cells and for whom Post‐Cy was used for GVHD prophylaxis. Results Myeloid reconstitution was achieved on day + 30 for 57 of the 62 patients. The median follow‐up of the surviving 39 patients (63%) was 26 months, with a range of 6‐57 months. The OS and EFS at 2 years were 64.6% (52.0%‐77.2%, 95% CI) and 58.9% (46.1%‐71.7%, 95% CI), respectively. The only factor in our multivariate analysis that contributed to an inferior EFS was a poor remission status prior to HSCT (HR, 8.30; 1.08‐63.56; P = 0.041, 95% CI). Conclusion The results of T‐cell replete haploidentical transplantation with Post‐Cy GVHD prophylaxis in high‐risk pediatric patients are promising. However, further research is needed to determine the factors that have affect HLA compatibility for predicting the success of haploidentical transplantations.