Trials--and errors.
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Biomedical subjects
Publications and source records attributed to D Foote.
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Hydroxyurea (HU) and sodium phenylbutyrate (SPB) have been shown to increase fetal hemoglobin (Hb F) levels in patients with thalassemia intermedia. The reported effects of these agents in increasing total Hb, however, have been inconsistent and there have been no studies on the combination of these medications. We describe the clinical response, as determined by increases in total Hb and decreased transfusion needs, in five patients with thalassemia intermedia treated with HU alone or in combination with SPB. All of the patients responded with increased levels of Hb F, but the responses in total Hb varied. Of the five patients, two had a marked response in total Hb in excess of 3 g/dl, two responded modestly with an increase in total Hb of 1-2 g/dl, and one did not respond. Prolonged responses were achieved with low doses of HU (3-10 mg/kg/day) and higher doses were associated with mild reversible hematologic or hepatic toxicity and no further increases in Hb. Sodium phenylbutyrate was added to treatment with HU in two patients, but failed to produce an increase in total Hb despite increasing Hb F levels. Of the four patients who responded to HU with an increase in total Hb, all reported symptomatic improvement and three have not required further transfusions. We conclude that low-dose HU therapy in patients with thalassemia intermedia may increase total Hb levels sufficiently to eliminate the need for transfusions. We, therefore, recommend a trial of HU for thalassemia intermedia patients in whom chronic transfusion therapy is being contemplated.
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Advances in diagnosis, medical management and surgical intervention have improved the longevity and quality of life for children with congenital heart disease. Despite this, research studies specifically examining the psychosocial concerns of adolescents and young adults with congenital heart disease are few. To explore the subjective experiences and dilemmas of this population during the transition from adolescence to young adulthood, we interviewed, using a semi-structured protocol, a convenience sample of nine adolescents and young adults. Using analytic procedures inherent in Grounded Theory methodology, seven themes were identified: the dilemma of normality; dilemmas in disclosure; dilemmas in strategies for management of illness; the challenge of social integration versus social isolation; the challenge of dependence versus independence; the challenge of uncertainty; and strategies for coping. An understanding of these experiences by health professionals can be beneficial in helping this clinical population as they grow up and face the challenges of an uncertain, yet promising, future.
Just a year ago most authorities considered the chances of patients contracting AIDS from doctors and other healthcare workers a virtual impossibility. But last week a Florida woman who got AIDS from her dentist lay near death, and two Minneapolis physicians admitted they had treated hundreds of patients since being diagnosed with the virus. Although doctors are at far greater risk than patients, the Minneapolis cases renewed the debate over the right of sides to know each other's HIV status.
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Diarrheal disease prevention and diarrheal disease control with oral rehydration therapy are major goals of the World Health Organization and the international health community. This paper promotes the use of ethnographic research in this endeavor. It illustrates the use of ethnography in a diarrheal disease prevention and control program in Honduras by illustrating how knowledge of local beliefs and practices partially shaped promotion efforts. It confirms the validity of the original ethnographic findings by reporting the results of a survey conducted two years after program onset.
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OBJECTIVE: To provide a better understanding of parents' experiences as their children with congenital heart disease mature through adolescence and young adulthood. DESIGN: A qualitative pilot study. SETTING: The physician practices of the pediatric cardiology service of a large university medical center. SUBJECTS: Eight parents of adolescents and young adults with congenital heart disease. INTERVENTION: Each parent was separately interviewed with use of a semistructured interview guide. RESULTS: Our study has identified seven themes--the dilemmas of normality, disclosure dilemmas, the challenge of uncertainty, illness management dilemmas and strategies, social integration versus social isolation, the impact of illness on the family, and coping--with which parents have struggled through-out their adolescent's and young adult's life. It was not possible to determine whether the experiences described by these parents are unique. CONCLUSIONS: Parents experience distress, as outlined in the seven themes. They need assistance to determine what is "normal" for their child and how to monitor their child's health and safety. Further research is needed to develop specific interventions.
Only a few long-term survivors of homozygous alpha-thalassemia, a usually fatal condition, have been reported. The authors present a surviving infant with this disorder and discuss the complications, treatments, and implications of this genetic hemoglobinopathy. The child had no antenatal intervention and has been treated with regular transfusions. She has had normal growth and development and is currently 2.5-years-old. A literature review of survivors with Bart hemoglobinopathy reveals an intense perinatal course and a great prevalence of congenital urogenital and limb defects. Advances in antenatal diagnosis, intrauterine intervention, and postnatal treatments have resulted in extended survival of children with congenital defects that until recently were considered invariably fatal. Transfusion and chelation therapy and bone marrow transplantation provide long-term treatment and potential curative options.