We reviewed the results of transplantation of allogeneic marrow from HLA-identical donors in patients with beta-thalassemia who were less than 16 years old. Among the 222 consecutive patients who had received transplants since 1983, survival and event-free-survival curves leveled off about one year after transplantation, at 82 and 75 percent, respectively. Pretransplantation clinical characteristics were examined for their impact on survival, event-free survival, and the recurrence of thalassemia in the 116 consecutive patients who were treated with our current regimen, in use since June 1985. In a multivariate analysis, portal fibrosis and either the presence of hepatomegaly or a history of inadequate chelation therapy were significantly associated with reduced probabilities of survival and event-free survival. The patients were divided into three classes on the basis of the presence of hepatomegaly or portal fibrosis (class 1 had neither factor, class 2 had one, and class 3 had both). For class 1 patients the three-year probabilities of survival, event-free survival, and recurrence were 94, 94, and 0 percent, respectively. For class 2 patients the probabilities were 80, 77, and 9 percent, and for class 3 patients 61, 53, and 16 percent. We conclude that for patients under 16 years of age, transplantation of bone marrow from an HLA-identical donor offers a high probability of complication-free survival, particularly if they do not have hepatomegaly or portal fibrosis.
The internal consistency, validity and factor structure of the 12-item General Health Questionnaire (GHQ-12) were investigated in a homogeneous sample consisting of 18-year-old males in Italy. The GHQ-12 proved to be a reliable instrument, as indicated by a Cronbach's alpha of 0.81. When the screening characteristics of the GHQ-12 (scored by the Likert method) were evaluated against the psychiatrist's ratings, the best balance between sensitivity and specificity was found at the GHQ cut-off score of 8/9: at this threshold, sensitivity was 0.68 and was paired to a specificity of 0.59 and an overall misclassification rate of 0.40. Validity coefficients based on a single severity score were rather low compared with those reported in other settings. When a principal components analysis with varimax (and oblimin) rotation was performed, two factors were identified: factor A (general dysphoria) was defined by 7 items related to anxiety and depression; factor B (social dysfunction) included 6 items testing the ability to perform daily activities and to cope with everyday problems. The identified factors revealed distinct ability in the discrimination between subjects with and without emotional disturbance according to the psychiatrist's ratings and correlated differently with 3 Minnesota Multiphasic Personality Inventory subscales (depression, D; conversion hysteria, Hy; psychasthenia, Pt). Thus, the factor structure of the GHQ-12 might provide useful information along with that offered by a single severity score, and the detection of cases might be improved by examining an individual's profile of scores on different subscales derived from factor analysis.
The usefulness of current psychiatric classification, which is based on ICD/DSM categorical diagnoses, remains questionable. A promising alternative has been put forward as the "transdiagnostic" approach. This is expected to cut across existing categorical diagnoses and go beyond them, to improve the way we classify and treat mental disorders. This systematic review explores whether self-defining transdiagnostic research meets such high expectations. A multi-step Web of Science literature search was performed according to an a priori protocol, to identify all studies that used the word "transdiagnostic" in their title, up to May 5, 2018. Empirical variables which indexed core characteristics were extracted, complemented by a bibliometric and conceptual analysis. A total of 111 studies were included. Most studies were investigating interventions, followed by cognition and psychological processes, and neuroscientific topics. Their samples ranged from 15 to 91,199 (median 148) participants, with a mean age from 10 to more than 60 (median 33) years. There were several methodological inconsistencies relating to the definition of the gold standard (DSM/ICD diagnoses), of the outcome measures and of the transdiagnostic approach. The quality of the studies was generally low and only a few findings were externally replicated. The majority of studies tested transdiagnostic features cutting across different diagnoses, and only a few tested new classification systems beyond the existing diagnoses. About one fifth of the studies were not transdiagnostic at all, because they investigated symptoms and not disorders, a single disorder, or because there was no diagnostic information. The bibliometric analysis revealed that transdiagnostic research largely restricted its focus to anxiety and depressive disorders. The conceptual analysis showed that transdiagnostic research is grounded more on rediscoveries than on true innovations, and that it is affected by some conceptual biases. To date, transdiagnostic approaches have not delivered a credible paradigm shift that can impact classification and clinical care. Practical "TRANSD"iagnostic recommendations are proposed here to guide future research in this field.
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