Endocrine Abstracts (2017) 49 EP887 | DOI: 10.1530/endoabs.49.EP887

Somatropin treatment Supported by NHS: characterization of submitted patients - 2006 to 2016

Margarida Bastos1,2, Alice Mirante1,2, Caldas Afonso1,3, Carlos Vasconcelos1,4, Conceição Bacelar1,3, Conceição Pereira1,5, Lurdes Lopes1,6, Marcelo Fonseca1,7, Joana Serra-Caetano2, Miguel Patrício1,2, Cesar Marques Esteves1,11, Florbela Ferreira1,8, Graciete Bragança1,9, Luísa Raimundo1,10, Lurdes Matos1,6, Lurdes Sampaio1,8, Manuel Fontoura1,11 & Teresa Borges1,3

1Comissão Nacional para a Normalização da Hormona de Crescimento (CNNHC), Portugal, Portugal; 2Endocrinology and Pediatric Endocrinology Departments of: CH Universitário de Coimbra, Coimbra, Portugal; 3CH Porto, Porto, Portugal; 4CH Lisboa Ocidental, Lisboa, Portugal; 5Oncology Portuguese Institute Francisco Gentil, Lisboa, Portugal; 6CH Lisboa Central, Lisboa, Portugal; 7Unidade ULSM, Matosinhos, Portugal; 8CH Lisboa Norte, Lisboa, Portugal; 9H Fernando Fonseca, Amadora, Portugal; 10CH Garcia de Horta, Almada, Portugal; 11CH S. João, Porto, Portugal; 12L. Biostatistics and MI and IBILI, FMC, U Coimbra, Coimbra, Portugal.

Introduction: In our country somatropin treatment is supported by the National Health Service. A National Committee (CNNHC) rules and analysis the submission papers of patients with: isolated/multiple somatotropin deficiency (STD), short stature in: renal chronic disease (DRC), small for gestational age (SGA), Turner syndrome (TS) and Prader Willi syndrome (PWS). In adults only isolated somatropin deficiency diagnosed in childhood.

Aims: To analyze the characteristics of patients submitted to the CNNHC from 2006 to 2016.

Methods: Retrospective study of cases submitted to CNNHC for somatotropin (2006–2016). Data collected: demographic, submission date, diagnosis and committee’s decision. Statistic analysis by SPSS21 (P<0.05).

Results: Total submissions n=1968 corresponding to 1909 children/six adults; Males (59.5%) and Females (40.5%). Mainly coming from Pediatric centres (n=1573/80.3%). Submissions increased along time, with a minimum of 87 in 2007 and a maximum of 252 in 2011. Were approved a total of n=1412 (72%) cases: at first submission n=1243 (63.4%) and after reevaluation n=169 (8.6%). Not approved n=535 (27.2%). Diagnostic prevalence: somatotropin deficiency n=1233 (62.9%): isolated n=1067(54.4%) and multiple n=166(8.5%); SGA n=324(16.5%), TS n=177(9%); RCD n=122(6.2%) and PWS n=53(2.7%). Somatotropin deficiency remained the most frequent diagnosis along the years. Age at submission: DRC were submitted earlier (6.4±4.2y), followed by SGA (7.9±3.0y), TS (8.5±3.6y), PWS (9.4±4.0y), multiple STD (9.7±5.1y) and isolated STD (10.3±3.5y). Except from TS, male gender was more frequent at all diagnosis. Were associated with oncologic disease n=182/1968(9.2%): n=97/182(53%) had primary tumours of CNS and n=66/182(36%) due to hemato-oncologic disease (leukemia/lymphoma).

Conclusions: Somatotropin supported treatment in our country has evolved along time, with new approved indications after 2010. The most frequent diagnosis remains isolated somatropin deficiency with a high age at submission witch probably compromises the final stature. We must have more support for deficient somatropin adult patients.