Growth hormone treatment of patients with neurofibromatosis type 1

K Haas-Lude; C Nagel; CP Schwarze; VF Mautner

doi:10.1055/s-0030-1265578

Neuropediatrics, Table of Contents

Growth hormone treatment of patients with neurofibromatosis type 1

K Haas-Lude ¹, C Nagel ², CP Schwarze ³, VF Mautner ⁴

¹Department of Pediatric Neurology, University Children's Hospital Tübingen
²Section of Pediatric Neurosurgery, Department of Neurosurgery, University Hospital Tübingen
³Section of Pediatric Endocrinology, University Children's Hospital Tübingen
⁴Section of Phacomatosis, Department of Oral and Maxillofacial Surgery, University Hospital Hamburg-Eppendorf

Abstract

Introduction: Short stature occurs in 20–30% of patients with neurofibromatosis type 1 (NF1), but growth hormone deficiency (GHD) is found only in few patients resulting from intracranial tumors, after cranial irradiation or cause idiopathic. Patients with NF1 have an increased risk of benign and malignant tumors.

Large cohort studies in tumor patients with growth hormone treatment (GHT) show that GHT is beneficial and without increased risk of tumor recurrence. There is no evidence that this is not true for NF1 patients. Nevertheless GHT in NF1 patients is still controversial. Because of the lack of prospective and randomized studies concerning risk and safety of GHT in this population recommendations from the respective societies do not exist.

Objective: The aim of our retrospective investigation was to evaluate a) the development of CNS and second tumors and changes of T2 weighted hyperintensities and b) the development of cutaneous neurofibromas during or after GHT in a small group of NF1 patients.

Results: Ten patients with NF1 were recorded. Seven subjects had idiopathic GHD, two were treated for chiasm glioma, and one presented a suspected low risk brainstem glioma. One of the chiasm glioma patients had a progress of the tumor during GHT, the other showed a resolution. No second tumors were detected. T2-weighted hyperintensities demonstrated the characteristics expected in the respective age groups. No cutaneous neurofibromas were found.

Conclusion: The data presented here and from the literature are not sufficient to estimate the risk of GH for NF1 patients concerning CNS tumor progress, development of second tumors and cutaneous neurofibromas compared to a non treated NF1 patient group. A prospective controlled study is therefore needed. A strictly negative view against GHT for patients with NF1 cannot be supported by our data.