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.

WNT signaling has been implicated in the regulation of hematopoietic stem cells and plays an important role during T-cell development in thymus. Here we investigated WNT pathway activation in childhood T-cell acute lymphoblastic leukemia (T-ALL) patients. To evaluate the potential role of WNT signaling in T-cell leukomogenesis, we performed expression analysis of key components of WNT pathway. More than 85% of the childhood T-ALL patients showed upregulated β-catenin expression at the protein level compared with normal human thymocytes. The impact of this upregulation was reflected in high expression of known target genes (AXIN2, c-MYC, TCF1 and LEF). Especially AXIN2, the universal target gene of WNT pathway, was upregulated at both mRNA and protein levels in ∼40% of the patients. When β-CATENIN gene was silenced by small interfering RNA, the cancer cells showed higher rates of apoptosis. These results demonstrate that abnormal WNT signaling activation occurs in a significant fraction of human T-ALL cases independent of known T-ALL risk factors. We conclude that deregulated WNT signaling is a novel oncogenic event in childhood T-ALL.

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The purpose of this study was to compare the efficacy, safety, and cost of piperacillin/tazobactam with cefepime monotherapy in children with febrile neutropenia. A prospective randomized study in children and adolescent with cancer was conducted. Patients were randomly assigned to receive either 80 mg/kg piperacillin/10 mg/kg tazobactam every 6 h (maximum 4.5 g/dose) or cefepime 50 mg/kg every 8 h (maximum 2 g/dose). Treatment modification was defined as all the changes in the empirical antimicrobials after the first 96 h. Overall treatment success was defined as cure of febrile episode with or without modification. Cost of hospitalization, antimicrobial drugs, and supportive therapy were calculated. Fifty febrile neutropenic episodes (25 in the piperacillin/tazobactam group, 25 in the cefepime group) in 27 pediatric cancer patients were evaluated. The groups were comparable in terms of age, gender, body weight, primary diagnosis, disease status, initial neutrophil count, and duration of neutropenia. Microbiologically and clinically documented infection rate was 46%. There was no infection-related mortality in the study period. The treatment success of initial empirical therapy without modification was not different in the 2 groups (56% in piperacillin/tazobactam group and 48% in cefepime group). Anti-anaerobic drugs were added more frequently in the cefepime group. Duration of fever, neutropenia, treatment, and cost of therapy were not different in the treatment groups. Piperacillin/tazobactam monotherapy is as effective as cefepime monotherapy in febrile neutropenia of pediatric cancer patients.

ObjectiveLoss of immunity to previous vaccination and timing of re‐vaccination in children receiving chemotherapy remains controversial. The aim of this study was to investigate the immunity to vaccine preventable diseases in children with acute lymphoblastic leukemia (ALL).ProcedureSixty‐one patients with ALL and 13 healthy siblings were enrolled. Three study groups included newly diagnosed patients (group 1), patients on maintenance chemotherapy (group 2), and patients that completed chemotherapy (group 3). Blood samples for baseline antibody titers were obtained from all the patients and controls. Patients in group 2 were vaccinated with diphtheria, tetanus, and hemophilus influenzae type b (Hib). Patients in group 3 and controls received the measles vaccine in addition to all the above vaccines. In groups 2 and 3, post‐vaccination antibody titers were also obtained.ResultsPatients and controls had no Hib vaccine during primary vaccination. After chemotherapy median antibody levels against diphtheria, tetanus, measles, and Hib were decreased but tetanus antibodies were still at the protective levels. Proportions of the patients with protective levels were 11.1%, 83.3%, 16.7%, and 16.7% for diphtheria, tetanus, Hib, and measles, respectively. Vaccination achieved protective antibody levels in 81%, 100%, 89.5%, and 70% of the patients for diphtheria, tetanus, Hib, and measles, respectively. Vaccine responses during maintenance were also satisfying.ConclusionWe recommend re‐vaccination after 3 months of cessation of chemotherapy. Administration of Hib vaccine may be beneficial after the first 3 months of maintenance chemotherapy especially in children with no primary vaccination followed by a second booster dose after cessation of therapy to increase immunity. Pediatr Blood Cancer 2009;53:967–972. © 2009 Wiley‐Liss, Inc.