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Growth Hormone Replacement Therapy in Children With Medulloblastoma: Use and Effect on Tumor Control

From the Departments of Neurology and Pediatrics, Children’s National Medical Center, The George Washington University, Washington, DC; Departments of Biosatistics and Neuro-Oncology, St Jude Children’s Research Hospital, Memphis, TN; Departments of Oncology, Neurology, and Endocrinology, Children’s Hospital of Philadelphia, Philadelphia, PA; Department of Neurosurgery, New York University, New York; Children’s Hospital of Buffalo and Roswell Park Cancer Institute, Buffalo, NY; Department of Neurosurgery, University of California at San Francisco, San Francisco; Children’s Hospital of Los Angeles, Los Angeles, CA; Children’s Memorial Hospital, Chicago, IL; Department of Pediatrics, Children’s Hospital and Regional Medical Center, Seattle, WA; Dana-Farber Cancer Institute, Boston, MA; Hospital for Sick Children, Toronto, Canada.

Address reprint requests to Roger J. Packer, MD, Department of Neurology, Children’s National Medical Center, 111 Michigan Avenue, NW, Washington, DC 20010; email rpacker{at}cnmc.org

PURPOSE: Progress has been made in the treatment of medulloblastoma, the most common childhood malignant brain tumor: However, many long-term survivors will have posttherapy growth hormone insufficiency with resultant linear growth retardation. Growth hormone replacement therapy (GHRT) may significantly improve growth, but there is often reluctance to initiate GHRT because of concerns of an increased likelihood of tumor relapse.

PATIENTS AND METHODS: This study retrospectively reviewed the use of GHRT for survivors of medulloblastoma in 11 neuro-oncology centers in North America who received initial treatment for disease between 1980 and 1993 to determine its impact on disease control. A Landmark analysis was used to evaluate the relative risk of relapse in surviving patients.

RESULTS: Five hundred forty-five consecutive patients less than 15 years of age at diagnosis were identified. Six-year progression-free survival (mean ± SD) was 40% ± 5% in children less than 3 years of age at diagnosis compared with 59% ± 3% for older patients. Older patients with total or near-total resections (P = .003) and localized disease at diagnosis (P < .0001) had the highest likelihood of survival. One hundred seventy patients (33% ± 3% of the cohort) received GHRT. GHRT use varied widely among institutions, ranging from 5% to 73%. GHRT was begun a mean of 3.9 years after diagnosis, later in children younger than 3 years at diagnosis (5.4 years). By Landmark analyses, for those surviving 2, 3, and 5 years after diagnosis, there was no evidence that GHRT increased the rate of disease relapse.

CONCLUSION: This large retrospective review demonstrates that GHRT is underutilized in survivors of medulloblastoma and is used relatively late in the course of the illness. GHRT is not associated with an increased likelihood of disease relapse.

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