Hemophilia Growth and Development Study: Design, Methods... : Journal of Pediatric Hematology/Oncology (original) (raw)

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Design, Methods Entry Data

Hilgartner, Margaret W. M.D.; Donfield, Sharyne M. Ph.D.; Willoughby, Anne M.D., M.P.H.; Contant, Charles F. Jr. Ph.D.; Evatt, Bruce L. M.D.; Gomperts, Edward D. M.D.; Hoots, W. Keith M.D.; Jason, Janine M.D.; Loveland, Katherine A. Ph.D.; McKinlay, Sonja M. Ph.D.; Stehbens, James A. Ph.D.

Division of Pediatric Hematology and Oncology, New York Hospital-Cornell Medical Center, New York (M.W.H.); New England Research Institute, Watertown, Massachusetts (S.M.D., S.M.M.); National Institute of Child Health and Human Development, Pediatric Adolescent and Maternal AIDS Branch, Bethesda, Maryland (A.W.); Department of Neurosurgery, Baylor College of Medicine, Houston, Texas (C.F.C.); National AIDS Information and Education Program (J.J.), Division of HIV/AIDS, Centers for Disease Control, Atlanta, Georgia (B.L.E.); Children's Hospital of Los Angeles, California (E.D.G.); Departments of Pediatrics and Internal Medicine, University of Texas Medical School at Houston, Houston, Texas (W.K.H.); Departments of Psychiatry and Behavioral Sciences, University of Texas Medical School, Houston, Texas (K.A.L.); and University of Iowa, Iowa City, Iowa (J.A.S.), U.S.A.

Abstract

Purpose

The study design, research questions, and baseline data are presented from a multicenter longitudinal epidemiologic investigation of the impact of human immunodeficiency virus (HIV) infection on three areas of functioning in children and adolescents with hemophilia: physical growth and sexual maturation, immune function, and neurological and neuropsychological functioning.

Patients and Methods

Sixty-nine percent (n = 333) of a population of males between the ages of 6 and 19 years with moderate to severe hemophilia participated in a comprehensive baseline examination. Approximately 62% of the study group was HIV seropositive and 38% HIV seronegative.

Results

Adjusted for age, HIV-positive participants were three times as likely as HIV-negative participants to exhibit declines in height-for-age attainment (p = 0.05), twice as likely to have delays in sexual maturation (p = 0.414), and more than three times as likely to exhibit antibody levels that were not indicative of prior exposure or vaccination (p < 0.001). In addition, analysis of a comprehensive battery of neuropsychological tests showed that HIV-positive participants were 50% more likely to show scores 1 SD below expected levels in three of nine functional areas (p = NS) compared with HIV-negative participants.

Conclusions

Entry data for this study reveal a greater likelihood for HIV-positive children and adolescents with moderate to severe hemophilia to have abnormal growth, and to exhibit antibody levels not indicative of prior exposure to disease or vaccination, which could be attributed to HIV infection. Lowered neuropsychological test performance in both groups may be attributable to the deleterious effects of chronic illness. Data are being collected from a group of non-HIV infected non-hemophiliac siblings for comparison.