Background Type 3 von Willebrand's disease (VWD) patients present markedly reduced levels of von Willebrand factor and factor VIII. Because of its rarity, the bleeding phenotype of type 3 VWD is poorly described, as compared to type 1 VWD. Aims To evaluate the frequency and the severity of bleeding symptoms across age and sex groups in type 3 patients and to compare these with those observed in type 1 VWD patients to investigate any possible clustering of bleeding symptoms within type 3 patients. Methods We compared the bleeding phenotype and computed the bleeding score (BS) using the MCMDM‐1VWD bleeding questionnaire in patients enrolled in the 3WINTERS‐IPS and MCMDM‐1VWD studies. Results In 223 unrelated type 3 VWD patients, both the BS and the number of clinically relevant bleeding symptoms were increased in type 3 as compared to type 1 VWD patients (15 versus 6 and 5 versus 3). Intracranial bleeding, oral cavity, hemarthroses, and deep hematomas were at least five‐fold over‐represented in type 3 VWD. A more severe bleeding phenotype was evident in patients having von Willebrand factor antigen levels < 20 IU/dL at diagnosis in the two merged cohorts. In type 3 patients, there was an apparent clustering of hemarthrosis with gastrointestinal bleeding and epistaxis, whereas bleeding after surgery or tooth extraction clusters with oral bleeding and menorrhagia. Conclusions In the largest cohort of type 3 VWD patients, we were able to describe a distinct clinical phenotype that is associated with the presence of a more severe hemostatic defect.

Background:Thalassemia is an inherited blood disease. It is a serious public health problem throughout the Mediterranean region, the Middle East and the Indian subcontinent, as well as in Southeast Asia.Objectives:Thalassemia is an inherited blood disease. It is a serious public health problem. In this study we assessed psychological aspects in Iranian children and adolescents with thalassemia major.Patients and Methods:In this case-control study sixty healthy subjects aged 7-18 years and Sixty Patients with confirmed diagnosis of major thalassemia were enrolled. After obtaining informed consent from parents of all participating thalassemia patients and healthycontrols, we assessed psychological aspects and quality of life by Pediatric Quality of LifeTM (PedsQL™), Strengths and Difficulties Questionnaires (SDQ), State and Trait Anxiety, Children's Depression Inventory (CDI). Results:The results of this study indicate that there are significant changes in depression, anxiety, QOL and behavioral screening between children with thalassemia major compared with healthy subjects by means of both parents and children reports. According to the results, children with thalassemia major have more psychological problems than healthy ones. Patients with thalassemia have a lower QOL than their peers (P = 0.001), the rate of depression is higher in this group (P = 0.015), Also behavioral problems in these children are more than healthy subjects (P = 0.009).Conclusions:We recommend appropriate treatment and counseling procedures in addition to specific treatment of thalassemia. According to the results we suggest to establish pediatric psychiatric clinics beside thalassemic clinics to cure psychological aspects of the disease.

Type 3 von Willebrand disease (VWD3) is a rare and severe bleeding disorder characterized by often undetectable von Willebrand factor (VWF) plasma levels, a recessive inheritance pattern, and heterogeneous genotype. The objective of this study was to identify the VWF defects in 265 European and Iranian patients with VWD3 enrolled in 3WINTERS-IPS (Type 3 Von Willebrand International Registries Inhibitor Prospective Study). All analyses were performed in centralized laboratories. The VWF genotype was studied in 231 patients with available DNA (121 [115 families] from Europe [EU], and 110 [91 families] from Iran [IR]). Among 206 unrelated patients, 134 were homozygous (EU/IR = 57/77) and 50 were compound heterozygous (EU/IR = 43/7) for VWF variants. In 22 patients, no or only one variant was found. A total of 154 different VWF variants (EU/IR = 101/58 [5 shared]) were identified among the 379 affected alleles (EU/IR = 210/169), of which 48 (EU/IR = 18/30) were novel. The variants p.Arg1659*, p.Arg1853*, p.Arg2535*, p.Cys275Ser, and delEx1_Ex5 were found in both European and Iranian VWD3 patients. Sixty variants were identified only in a single allele (EU/IR = 50/10), whereas 18 were recurrent (≥3 patients) within 144 affected alleles. Nine large deletions and one large insertion were found. Although most variants predicted null alleles, 21% of patients carried at least 1 missense variant. VWD3 genotype was more heterogeneous in the European population than in the Iranian population, with nearly twice as many different variants. A higher number of novel variants were found in the Iranian VWD3 patients.

Khodashenas E, Badiee Z, Sohrabi M, Ghassemi A, Hosseinzade V. The effect of an aerobic exercise program on the quality of life in children with cancer. Turk J Pediatr 2017; 59: 678-683. Aerobic exercise such as physical activity plays an essential role for a healthy development for children undergoing chemotherapy and radiation therapy. The aim of this study was to examine the effectiveness of an aerobic exercise program on quality of life in children undergoing cancer treatment. Patients diagnosed with acute lymphoblastic leukemia (ALL), undergoing chemotherapy, were divided into two groups; the control group received the routine treatment with activities of daily living and the interventional group received routine treatment in addition to an aerobic exercise program. Data was collected using the pediatric quality of life questionnaires (PedsQL version 4) before and after the training program. We enrolled 20 cancer patients, who were referred to Dr. Sheikh Children`s Hospital in Mashhad, Iran; 8 (40%) and 12 (60%) of the patients were female and male, respectively. The results show a significant association in the pain and injury subscale (p=0.002), improving in interventional groups after test compared to the control groups. The reports of parents of the children`s quality of life showed significant association in the pain and injury subscale (P=0.002), and cognitive problems subscale at school (P=0.039). The regular exercise and psychosocial program intervention can improve wellbeing and thereby enhance efficacy of the children during treatment.

Background: Thalassemia is an inherited blood disease. It is a serious public health problem throughout the Mediterranean region, the Middle East and the Indian subcontinent, as well as in Southeast Asia. Objectives: Thalassemia is an inherited blood disease. It is a serious public health problem. In this study we assessed psychological aspects in Iranian children and adolescents with thalassemia major. Patients and Methods:In this case-control study sixty healthy subjects aged 7-18 years and Sixty Patients with confirmed diagnosis of major thalassemia were enrolled. After obtaining informed consent from parents of all participating thalassemia patients and healthycontrols, we assessed psychological aspects and quality of life by Pediatric Quality of LifeTM (PedsQL™), Strengths and Difficulties Questionnaires (SDQ), State and Trait Anxiety, Children's Depression Inventory (CDI). Results:The results of this study indicate that there are significant changes in depression, anxiety, QOL and behavioral screening between children with thalassemia major compared with healthy subjects by means of both parents and children reports. According to the results, children with thalassemia major have more psychological problems than healthy ones. Patients with thalassemia have a lower QOL than their peers (P = 0.001), the rate of depression is higher in this group (P = 0.015), Also behavioral problems in these children are more than healthy subjects (P = 0.009). Conclusions:We recommend appropriate treatment and counseling procedures in addition to specific treatment of thalassemia. According to the results we suggest to establish pediatric psychiatric clinics beside thalassemic clinics to cure psychological aspects of the disease.