Translation and validation of the Turkish version of the Ankylosing Spondylitis Quality of Life (ASQOL) questionnaire.

The Ankylosing Spondylitis Quality of Life (ASQoL) questionnaire is a disease-specific measure of needs-based quality of life developed in the UK and the Netherlands. This study describes translation, validation, and reliability of the scale into Turkish population. The ASQoL was translated into Turkish using the dual-panel process. Content validity was assessed via cognitive debriefing interviews with ankylosing spondylitis (AS) patients. Patients with AS according to modified New York criteria were recruited into the study from 12 hospitals of all part of Turkey. Psychometric and scaling properties were assessed via a two administration survey involving the ASQoL, the Nottingham Health Profile (NHP), Bath AS Functional Index (BASFI), and Bath AS Disease Activity Index (BASDAI). Classical psychometrics assessed reliability, convergent validity (correlation of ASQoL with NHP, BASFI, and BASDAI) and discriminative validity (correlation of ASQoL with perceived AS-severity and general health). Cognitive debriefing showed the new Turkish ASQoL to be clear, relevant, and comprehensive. Completed survey questionnaires were received from 277 AS patients (80% Male, mean age 42.2/SD 11.6, mean AS duration 9.4 years/SD 9.4). Test-retest reliability was excellent (0.96), indicating low random measurement error for the scale. Correlations of ASQoL with NHP sections were low to moderate (NHP Sleep 0.34; NHP Emotional Reactions 0.83) suggesting the measures assess related but distinct constructs. The measure was able to discriminate between patients based on their perceived disease severity (p < 0.0001) and self-reported general health (p < 0.0001). The Turkish version of ASQoL has good reliability and validity properties. It is practical and useful scale to assess the quality of life in AS patients in Turkish population.

Thiamine-responsive megaloblastic anemia with diabetes mellitus and sensorineural deafness.

This study introduces a patient who has thiamine and thiamine pyrophosphokinase (TPKase) enzyme deficiency associated with diabetes mellitus, sensorineural deafness and thiamine-responsive megaloblastic anemia. Diabetes mellitus was diagnosed when she was 20 months old. After 1 year, macrocytic anemia developed and the thiamine therapy was started at 75 mg/day. During the follow-up, the insulin requirement decreased and even ceased, and macrocytic anemia improved with thiamine treatment. After thiamine therapy was ceased an increase in insulin requirement was observed and macrocytic anemia developed again.

The effect of high-dose methylprednisolone treatment on GM-CSF level in children with acute leukemia: a pilot study.

High-dose methylprednisolone therapy (HDMP) induces acceleration of leukocyte recovery in acute lymphoblastic leukemia (ALL) and the differentiation of myeloblasts to mature granulocytes in acute myeloblastic leukemia (AML). These effects of corticosteroids have been shown to be due to the enhanced colony-stimulating activity (CSA) and responses to corticosteroids in some patients with aplastic anemia and myelodysplastic syndromes (MDS) have been related to increased CSA activity. We measured the serum (granulocyte-macrophage colony-stimulating factor (GM-CSF) levels by a sandwich linked immunoabsorbent assay (ELISA) in patients with ALL and AML at presentation and following high-dose methylprednisolone (HDMP) therapy. Serum GM-CSF levels at presentation in the ten cases studied ranged between 160 and 700 pg/ml (mean 418.5 +/- 252.5). One week following HDMP therapy GM-CSF levels increased to between 260 and 950 pg/ml (733.5 +/- 203.2). Four weeks after therapy the GM-CSF levels increased to between 470 and 1350 pg/ml (911 +/- 278.7). GM-CSF levels were markedly elevated one week after HDMP in the patients with ALL, suggesting that in addition to the lymphotoxic effects on leukemic blasts, the acceleration in neutrophil recovery may be due to release of GM-CSF induced by HDMP and its effects on myeloid progenitors.

High remission rate in acute myeloblastic leukemia in children treated with high-dose methylprednisolone.

Since the differentiating effect of high-dose methylprednisolone (HDMP) on myeloid leukemic cells has been shown in one of our patients with acute myeloblastic leukemia (AML-M4), 27 previously untreated children with AML were given HDMP (20-30 mg/kg per day) combined with cytosine arabinoside (Ara-C; 3 mg/kg) for the first 2 weeks of induction therapy. Marked clinical improvement was observed in all patients with the exception of one who died within 24 hours of the treatment. Enlarged liver and spleen (greater than 5 cm) became nonpalpable in 3 (37%) out of 8 and 5 (100%) out of 5 patients, respectively, and bone marrow blasts decreased below 5% in 7 patients (27%) within 2 wk of HDMP and Ara-C treatment. Adriamycin (1 mg/kg) was added 2 wk after initiation of induction therapy. Twenty-two (84.6%) of the 26 patients achieved complete remission, 3 (11.5%) had partial remission and no response was obtained in one. Treatment was well tolerated. The addition of HDMP as a differentiating and/or cytolytic agent to conventional anti-leukemic chemotherapy increased the complete remission rate and prolonged the duration of remission of our AML patients.

Mega-dose intravenous methylprednisone for the treatment of onyalai: a case report.

Onyalai, a peculiar manifestation of idiopathic thrombocytopenic purpura characterized by a haemorrhagic bullae of the oral mucosa and lips, was diagnosed in a 1.5-month-old Turkish boy. He was treated with mega-dose intravenous methylprednisolone (30 mg/kg for 3 days) with prompt platelet response and improvement of oral mucosal findings.