Urania Dagalakis1, Maya Lodish2, Eva Dombi3, Ninet Sinaii4, Jessica Sabo3, Andrea Baldwin3, Seth M Steinberg5, Constantine A Stratakis1, Brigitte C Widemann3. 1. Section on Endocrinology and Genetics, Program on Developmental Endocrinology & Genetics, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD. 2. Section on Endocrinology and Genetics, Program on Developmental Endocrinology & Genetics, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD. Electronic address: lodishma@mail.nih.gov. 3. Pharmacology and Experimental Therapeutics Section, Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, Bethesda, MD. 4. Biostatistics and Clinical Epidemiology Service, National Institutes of Health Clinical Research Center, Bethesda, MD. 5. Biostatistics and Data Management Section, Center for Cancer Research, National Cancer Institute, Bethesda, MD.
Abstract
OBJECTIVE: To assess the relationship between pubertal progression and change in plexiform neurofibroma (PN) burden over time in pediatric and young adult patients with neurofibromatosis type 1 and PNs. STUDY DESIGN: Analyses accounted for sex, age, race, and chemotherapy. Forty-one patients with neurofibromatosis type 1 (15 female and 26 male patients) were studied at the National Institutes of Health. Tanner stage, testosterone, progesterone, estradiol, insulin-like growth factor -1, luteinizing hormone, and follicle-stimulating hormone were assessed. Tumor volume was measured using magnetic resonance imaging and lesion detection software developed locally. Patients were divided into 2 groups based on whether they were actively progressing through puberty (n = 16) or were peripubertal (n = 25) and were followed for an average of 20 months. Tumor growth rates in the puberty and peripubertal group were analyzed for a subset of patients. RESULTS: There was no statistically significant difference in tumor burden change over time (cm(2)/kg per month) between the pubertal and peripubertal groups (-0.16 ± 0.34 vs 0.03 ± 1.8, P = .31) and in the PN growth rates before and during puberty (P = .90). Change in tumor volume/patient weight/time did not correlate with testosterone change/time in males or estradiol change/time in females. CONCLUSION: These findings support that hormonal changes of puberty do not accelerate PN growth. Additional long-term follow-up of patients is necessary to further characterize the interaction between puberty and tumor growth. Published by Mosby, Inc.
OBJECTIVE: To assess the relationship between pubertal progression and change in plexiform neurofibroma (PN) burden over time in pediatric and young adult patients with neurofibromatosis type 1 and PNs. STUDY DESIGN: Analyses accounted for sex, age, race, and chemotherapy. Forty-one patients with neurofibromatosis type 1 (15 female and 26 male patients) were studied at the National Institutes of Health. Tanner stage, testosterone, progesterone, estradiol, insulin-like growth factor -1, luteinizing hormone, and follicle-stimulating hormone were assessed. Tumor volume was measured using magnetic resonance imaging and lesion detection software developed locally. Patients were divided into 2 groups based on whether they were actively progressing through puberty (n = 16) or were peripubertal (n = 25) and were followed for an average of 20 months. Tumor growth rates in the puberty and peripubertal group were analyzed for a subset of patients. RESULTS: There was no statistically significant difference in tumor burden change over time (cm(2)/kg per month) between the pubertal and peripubertal groups (-0.16 ± 0.34 vs 0.03 ± 1.8, P = .31) and in the PN growth rates before and during puberty (P = .90). Change in tumor volume/patient weight/time did not correlate with testosterone change/time in males or estradiol change/time in females. CONCLUSION: These findings support that hormonal changes of puberty do not accelerate PN growth. Additional long-term follow-up of patients is necessary to further characterize the interaction between puberty and tumor growth. Published by Mosby, Inc.
Authors: Rosa Nguyen; Eva Dombi; Brigitte C Widemann; Jeffrey Solomon; Carsten Fuensterer; Lan Kluwe; Jan M Friedman; Victor-Felix Mautner Journal: Orphanet J Rare Dis Date: 2012-10-04 Impact factor: 4.123
Authors: Sripriya Raman; Adda Grimberg; Steven G Waguespack; Bradley S Miller; Charles A Sklar; Lillian R Meacham; Briana C Patterson Journal: J Clin Endocrinol Metab Date: 2015-04-03 Impact factor: 5.958
Authors: Robert A Avery; James A Katowitz; Michael J Fisher; Gena Heidary; Eva Dombi; Roger J Packer; Brigitte C Widemann Journal: Ophthalmology Date: 2016-11-03 Impact factor: 12.079