ESPE2023 Poster Category 1 Growth and Syndromes (75 abstracts)
PROGRES, a multi-country, non-interventional, prospective study of patients receiving human growth hormone treatment under routine clinical care: Study update
Mitchell Geffner 1 , Aristides Maniatis 2 , Lourdes Ibañez 3 , Daria La Torre 4 , Carol Huang 5 , Feyza Darendeliler 6 , Mehul Dattani 7 , Mohamad Maghnie 8,9 , Moshe Phillip 10 , Reiko Horikawa 11 , Roy Gomez 12 , Shilpa Viswanathan 13 , Martin Carlsson 13 & Michael Wajnrajch 13,14
1The Saban Research Institute, Children’s Hospital Los Angeles, Keck School of Medicine of the University of Southern California, Los Angeles, USA. 2Rocky Mountain Pediatric Endocrinology, Centennial, USA. 3Endocrinology, Institut de Recerca Sant Joan de Déu, University of Barcelona, Barcelona, Spain. 4Pfizer, Rome, Italy. 5Alberta Children’s Hospital, Calgary, Canada. 6İstanbul Faculty of Medicine, Pediatric Endocrinology Unit, İstanbul, Turkey. 7UCL Great Ormond Street Institute of Child Health, London, United Kingdom. 8Clinica Pediatrica, IRCCS Istituto Giannina Gaslini, Genoa, Italy. 9DINOGMI, University of Genoa, Genoa, Italy. 10Schneider Children’s Medical Center of Israel, Petah Tikva, and Sackler Faculty of Medicine, Tel-Aviv University, Tel-Aviv, Israel. 11National Center for Child Health and Development, Tokyo, Japan. 12Pfizer Pte Ltd, Singapore, Singapore. 13Pfizer Inc, New York, USA. 14New York University, Grossman School of Medicine, New York, USA
Objectives: Children with growth hormone deficiency (GHD) are usually treated with once-daily injections of recombinant human growth hormone (rhGH). Somatrogon is a long-acting rhGH (LAGH) approved in the EU and other countries for once-weekly treatment of children with short stature. The Pfizer Registry of Outcomes in Growth hormone RESearch (PROGRES) study will assess the long-term safety and effectiveness of once-weekly somatrogon and once-daily rhGH preparations for treating children with growth abnormalities under routine clinical care; PROGRES will also enable comparisons across different once-daily rhGH preparations.
Methods: This non-interventional, prospective, phase 4 study aims to enroll eligible patients (male/female; any age) from >20 countries. Enrollment is from September 2021-October 2029, with data collected until October 2030 (minimum 1-year follow-up). Study inclusion criteria include prescription of once-weekly somatrogon or one of the daily rhGH preparations (Genotropin, Norditropin, Humatrope, or Omnitrope) and provision of informed consent. Somatrogon-treated patients are eligible for inclusion if somatrogon is approved and commercially available in their country. Primary safety outcomes include adverse events (AEs), serious AEs, and AEs of special interest. Primary effectiveness outcomes include annual height velocity (HV) and change in HV standard deviation scores (SDS) from baseline.
Results: One hundred and thirty-eight patients were enrolled as of 31 March 2023, with idiopathic GHD the most common primary diagnosis in all treatment groups (Table). Most patients are from Japan (majority receiving Genotropin) or the USA (majority receiving another daily rhGH). Overall, the mean age (SD) of patients was 12.1 (2.59) years and 23.2% were female; mean height SDS and BMI SDS (SD) at enrollment were -1.16 (0.95) and -0.14 (0.91), respectively.
| Somatrogon (n=2) | Genotropin (n=97) | Other daily rhGH (Norditropin, Humatrope, or Omnitrope) (n=39) | |
| Age, mean (SD), years | 11.8 (0.59) | 12.5 (2.45) | 11.2 (2.81) |
| Female, n (%) | 0 | 18 (18.6) | 14 (35.9) |
| Country | |||
| Australia | 0 | 1 (1.0) | 0 |
| France | 0 | 7 (7.2) | 3 (7.7) |
| Japan | 2 (100.0) | 65 (67.0) | 4 (10.3) |
| United Kingdom | 0 | 0 | 2 (5.1) |
| USA | 0 | 24 (24.7) | 30 (76.9) |
| Height SDS | |||
| n | 2 | 92 | 38 |
| Mean (SD) | -1.92 (0.15) | -1.33 (0.82) | -0.71 (1.11) |
| BMI SDS | |||
| n | 2 | 92 | 38 |
| Mean (SD) | 0.37 (0.65) | -0.17 (0.89) | -0.09 (0.99) |
| Primary diagnosis | |||
| Idiopathic GHD | 2 (100.0) | 87 (89.7) | 29 (74.4) |
| Prader-Willi Syndrome | 0 | 5 (5.2) | 0 |
| Other | 0 | 5 (5.2) | 10 (25.6) |
Conclusions: To date, 138 patients from 5 countries are enrolled in PROGRES, most having a primary diagnosis of idiopathic GHD.
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