Antonella Lonero1, Massimo Giotta2, Giulia Guerrini3, Valeria Calcaterra4,5, Elena Galazzi6, Lorenzo Iughetti7, Alessandra Cassio8,9, Gabriela Malgorzata Wasniewska10, Chiara Mameli5, Gianluca Tornese11, Mariacarolina Salerno12, Valentino Cherubini13, Manuela Caruso Nicoletti14, Maria Elisabeth Street15,16, Anna Grandone17, Claudio Giacomozzi18, Maria Felicia Faienza19, Chiara Guzzetti20, Simonetta Bellone21, Maria Parpagnoli22, Gianluca Musolino23, Maria Cristina Maggio24, Mauro Bozzola25, Paolo Trerotoli2, Maurizio Delvecchio26. 1. Giovanni XXIII Children's Hospital, AOU Policlinico di Bari, piazza G.Cesare 11, Bari, Italy. 2. Department of Biomedical Science and Human Oncology, University of Bari "Aldo Moro", Bari, Italy. 3. Specialty School of Paediatrics - Alma Mater Studiorum, University of Bologna, Bologna, Italy. 4. Pediatric and Adolescent Unit, Department of Internal Medicine, University of Pavia, Pavia, Italy. 5. Pediatric Department, "V. Buzzi" Children's Hospital, University of Milan, Milan, Italy. 6. Department of Endocrine and Metabolic Diseases, IRCCS Istituto Auxologico Italiano, Milan, Italy. 7. Department of Medical and Surgical Sciences of the Mother, Children and Adults, Pediatric Unit, University of Modena and Reggio Emilia, Modena, Italy. 8. Department of Pediatric Endocrinology, Unit of Pediatrics, University of Bologna, Bologna, Italy. 9. Department of Medical and Surgical Sciences, University of Bologna, Bologna, Italy. 10. Department of Human Pathology of Adulthood and Childhood, Unit of Pediatrics, University of Messina, Messina, Italy. 11. Institute for Maternal and Child Health, IRCCS Burlo Garofolo, Trieste, Italy. 12. Department of Translational Medical Sciences, Paediatric Endocrinology Unit, University of Naples 'Federico II', Naples, Italy. 13. Department of Women's and Children's Health, G. Salesi Hospital, Azienda Ospedaliero-Universitaria Ospedali Riuniti, Ancona, Italy. 14. Department of Paediatrics, "G Rodolico Hospital", University of Catania, Catania, Italy. 15. Department of Mother and Child, AUSL-IRCCS of Reggio Emilia, Reggio Emilia, Italy. 16. Department of Medicine and Surgery, University of Parma, Parma, Italy. 17. Department of Woman, Child and of General and Specialized Surgery, University of Campania "Luigi Vanvitelli", Naples, Italy. 18. Unit of Pediatrics, Department of Maternal and Child Health, Carlo Poma Hospital, ASST-Mantova, Mantua, Italy. 19. Department of Biomedical Sciences and Human Oncology, Paediatric Unit, University of Bari "A. Moro", Bari, Italy. 20. SSD Endocrinologia Pediatrica e Centro Screening Neonatale, Ospedale Pediatrico Microcitemico "A. Cao", ASSL Cagliari, Novara, Italy. 21. SCDU of Pediatrics, Department of Health Sciences, University of Piemonte Orientale, Novara, Italy. 22. Anna Meyer Children's Hospital, Florence, Italy. 23. Ambulatorio di Auxologia ed Endocrinologia pediatrica, S.C. Pediatria, Ospedale Pediatrico "Filippo Del Ponte", ASST Sette Laghi, Varese, Italy. 24. Department of Health Promotion, Maternal and Infantile Care, Department of Internal Medicine and Medical Specialties "G. D'Alessandro", University of Palermo, Palermo, Italy. 25. Il bambino e il suo pediatra-Onlus, Novara, Italy. 26. Giovanni XXIII Children's Hospital, AOU Policlinico di Bari, piazza G.Cesare 11, Bari, Italy. mdelvecchio75@gmail.com.
Abstract
PURPOSE: We aimed to evaluate the near-final height (nFHt) in a large cohort of pediatricpatients with growth hormone deficiency (GHD) and to elaborate a new predictive method of nFHt. METHODS: We recruited GHD patients diagnosed between 1987 and 2014 and followed-up until nFHt. To predict the values of nFHt, each predictor was run in a univariable spline. RESULTS: We enrolled 1051 patients. Pre-treatment height was -2.43 SDS, lower than parental height (THt) (-1.09 SDS, p < 0.001). The dose of recombinant human GH (rhGH) was 0.21mg/kg/week at start of treatment. nFHt was -1.08 SDS (height gain 1.27 SDS), higher than pre-treatment height (p < 0.001) and comparable to THt. 1.6% of the patients were shorter than -2 SDS from THt. The rhGH dose at nFHt was 0.19 mg/kg/week, lower than at the start (p < 0.001). The polynomial regression showed that nFHt was affected by gender, THt, age at puberty, height at puberty, age at the end of treatment (F = 325.37, p < 0.0001, R2 87.2%). CONCLUSION: This large national study shows that GHD children can reach their THt. The rhGH/kg/day dose significantly decreased from the start to the end of the treatment. Our model suggests the importance of a timely diagnosis, possibly before puberty, the beneficial effect of long-term treatment with rhGH, and the key-role of THt. Our prediction model has a very acceptable error compared to the majority of other published studies.
PURPOSE: We aimed to evaluate the near-final height (nFHt) in a large cohort of pediatricpatients with growth hormone deficiency (GHD) and to elaborate a new predictive method of nFHt. METHODS: We recruited GHD patients diagnosed between 1987 and 2014 and followed-up until nFHt. To predict the values of nFHt, each predictor was run in a univariable spline. RESULTS: We enrolled 1051 patients. Pre-treatment height was -2.43 SDS, lower than parental height (THt) (-1.09 SDS, p < 0.001). The dose of recombinant human GH (rhGH) was 0.21mg/kg/week at start of treatment. nFHt was -1.08 SDS (height gain 1.27 SDS), higher than pre-treatment height (p < 0.001) and comparable to THt. 1.6% of the patients were shorter than -2 SDS from THt. The rhGH dose at nFHt was 0.19 mg/kg/week, lower than at the start (p < 0.001). The polynomial regression showed that nFHt was affected by gender, THt, age at puberty, height at puberty, age at the end of treatment (F = 325.37, p < 0.0001, R2 87.2%). CONCLUSION: This large national study shows that GHD children can reach their THt. The rhGH/kg/day dose significantly decreased from the start to the end of the treatment. Our model suggests the importance of a timely diagnosis, possibly before puberty, the beneficial effect of long-term treatment with rhGH, and the key-role of THt. Our prediction model has a very acceptable error compared to the majority of other published studies.
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