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6. Development of a Measure to Assess Attitudes Towards Nasal versus Autoinjector Glucagon Delivery Devices for Treatment of Severe Hypoglycemia

Author

Bajpai, Sanjay K, primary, Cambron-Mellott, M Janelle, additional, Will, Oliver, additional, Poon, Jiat-Ling, additional, Wang, Qianqian, additional, Mitchell, Beth D, additional, Peck, Eugenia Y, additional, Babrowicz, Jane, additional, Raibulet, Nedina K, additional, Child, Christopher J, additional, and Beusterien, Kathleen, additional

Published
2022
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12. Development of a Measure to Assess Attitudes Towards Nasal versus Autoinjector Glucagon Delivery Devices for Treatment of Severe Hypoglycemia

Author

Bajpai,Sanjay K, Cambron-Mellott,M Janelle, Will,Oliver, Poon,Jiat-Ling, Wang,Qianqian, Mitchell,Beth D, Peck,Eugenia Y, Babrowicz,Jane, Raibulet,Nedina K, Child,Christopher J, Beusterien,Kathleen, Bajpai,Sanjay K, Cambron-Mellott,M Janelle, Will,Oliver, Poon,Jiat-Ling, Wang,Qianqian, Mitchell,Beth D, Peck,Eugenia Y, Babrowicz,Jane, Raibulet,Nedina K, Child,Christopher J, and Beusterien,Kathleen

Abstract

Sanjay K Bajpai,1 M Janelle Cambron-Mellott,2 Oliver Will,2 Jiat-Ling Poon,1 Qianqian Wang,1 Beth D Mitchell,1 Eugenia Y Peck,2 Jane Babrowicz,2 Nedina K Raibulet,1 Christopher J Child,1 Kathleen Beusterien2 1Eli Lilly and Company, Indianapolis, IN, USA; 2Cerner Enviza, New York, NY, USACorrespondence: Sanjay K Bajpai, Eli Lilly & Company, 893 S Delaware St, Indianapolis, IN, 46225, USA, Tel +1 317 931 9828, Email sanjay.bajpai@lilly.comBackground: For individuals managing diabetes, the administration of glucagon for severe hypoglycemia can be lifesaving, yet, until recently, there were no easy-to-use devices for these stressful emergencies. New products have emerged to meet this need, including nasal glucagon (NG) and auto-injector glucagon (AI). This study evaluated the psychometric properties of a new measure, the Glucagon Device Attitudes Questionnaire (GDAQ), in assessing attitudes toward NG and AI from the perspectives of persons with diabetes on insulin (PWDs), caregivers, and acquaintances.Methods: Developed based on qualitative research, the GDAQ consists of 38 rating items for each device and 16 direct-elicitation of attitudes of device relative to each other. It was administered to participants via a cross-sectional online survey. Twenty-six rating items were included in principal component analysis and confirmatory factor analysis. Items comprising each factor were averaged to form scales. Additionally, 12 direct elicitation items were averaged to form an overall “Attitudes” scale. Reliability and validity analyses were conducted. Descriptive statistics were provided for the rating items not included in the factor analysis.Results: A total of 405 PWDs, 313 caregivers, and 305 acquaintances participated. Three factors were identified: “Prepared and Protected” (7 items), “Hesitation” (12 items), and “Device Perceptions by Others” (7 items); factor loadings ranged from 0.13 to 0.92, 0.50 to 0.89, and 0.16 to 0.92, respectively. Cronbachâ

Published
2022

14. Producing a preference-based quality of LIFE measure to quantify the impact of HYPOGLYCAEMIA on people living with diabetes:A mixed-methods research protocol

Author

Carlton, Jill, Powell, Philip, Rowen, Donna, Broadley, Melanie, Pouwer, Frans, Speight, Jane, Heller, Simon, Gall, Mari-Anne, Rosilio, Myriam, Child, Christopher J, Comins, Jonathan, McCrimmon, Rory J, de Galan, Bastiaan, Brazier, John, Interne Geneeskunde, MUMC+: MA Endocrinologie (9), RS: Carim - V01 Vascular complications of diabetes and metabolic syndrome, and Medical psychology

Subjects
All institutes and research themes of the Radboud University Medical Center, Endocrinology, Endocrinology, Diabetes and Metabolism, Internal Medicine, Metabolic Disorders Radboud Institute for Molecular Life Sciences [Radboudumc 6]
Abstract

Contains fulltext : 291407.pdf (Publisher’s version ) (Open Access) BACKGROUND: Assessment of patient-reported outcome measures (PROMs), including quality of life (QoL), is essential in diabetes research and care. However, a recent review concluded that current hypoglycaemia-specific PROMs have limited evidence of validity, reliability and responsiveness for assessing the impact of hypoglycaemia on QoL in people living with diabetes. None of the PROMs identified could be used directly to inform the cost-effectiveness of treatments and interventions. There is a need for a new hypoglycaemia-specific QoL PROM, which can be used directly to inform economic evaluations. AIMS: This project has three aims: (a) To develop draft PROM content for measuring the impact of hypoglycaemia on QoL in adults with diabetes. (b) To refine the draft content using cognitive debriefing interviews and psychometrics. This will result in a condition-specific PROM that can be used to quantify the impact of hypoglycaemia upon QoL. (c) To generate a preference-based measure (PBM) that will enable utility values to be calculated for economic evaluation. METHODS: A mixed-methods, three-stage design is used: (a) Qualitative interviews will inform the draft PROM content. (b) Cognitive debriefing interview data will be used to refine the draft PROM content. The PROM will be administered in a large-scale survey to enable psychometric validation. Final item selection for the PROM will be informed by psychometric performance, translatability assessment and input from stakeholder groups. (c) A classification system will be generated, comprising a reduced number of items from the PROM. A valuation survey will be conducted to derive a value set for the PBM.

Published
2022
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15. Producing a preference‐based quality of LIFE measure to quantify the impact of HYPOGLYCAEMIA on people living with diabetes: A mixed‐methods research protocol.

Author

Carlton, Jill, Powell, Philip, Rowen, Donna, Broadley, Melanie, Pouwer, Frans, Speight, Jane, Heller, Simon, Gall, Mari‐Anne, Rosilio, Myriam, Child, Christopher J., Comins, Jonathan, McCrimmon, Rory J., de Galan, Bastiaan, and Brazier, John

Subjects
HEALTH outcome assessment, DIABETES, PATIENTS' attitudes, PSYCHOMETRICS, CONCEPTUAL structures, QUALITY of life, HYPOGLYCEMIA, COST effectiveness, RESEARCH funding
Abstract

Background: Assessment of patient‐reported outcome measures (PROMs), including quality of life (QoL), is essential in diabetes research and care. However, a recent review concluded that current hypoglycaemia‐specific PROMs have limited evidence of validity, reliability and responsiveness for assessing the impact of hypoglycaemia on QoL in people living with diabetes. None of the PROMs identified could be used directly to inform the cost‐effectiveness of treatments and interventions. There is a need for a new hypoglycaemia‐specific QoL PROM, which can be used directly to inform economic evaluations. Aims: This project has three aims: (a) To develop draft PROM content for measuring the impact of hypoglycaemia on QoL in adults with diabetes. (b) To refine the draft content using cognitive debriefing interviews and psychometrics. This will result in a condition‐specific PROM that can be used to quantify the impact of hypoglycaemia upon QoL. (c) To generate a preference‐based measure (PBM) that will enable utility values to be calculated for economic evaluation. Methods: A mixed‐methods, three‐stage design is used: (a) Qualitative interviews will inform the draft PROM content. (b) Cognitive debriefing interview data will be used to refine the draft PROM content. The PROM will be administered in a large‐scale survey to enable psychometric validation. Final item selection for the PROM will be informed by psychometric performance, translatability assessment and input from stakeholder groups. (c) A classification system will be generated, comprising a reduced number of items from the PROM. A valuation survey will be conducted to derive a value set for the PBM. [ABSTRACT FROM AUTHOR]

Published
2023
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19. Safety Outcomes During Pediatric GH Therapy: Final Results From the Prospective GeNeSIS Observational Program

Author

Child, Christopher J, Zimmermann, Alan G, Chrousos, George P, Cummings, Elisabeth, Deal, Cheri L, Hasegawa, Tomonobu, Jia, Nan, Lawrence, Sarah, Linglart, Agnès, Loche, Sandro, Maghnie, Mohamad, Pérez Sánchez, Jacobo, Polak, Michel, Predieri, Barbara, Richter-Unruh, Annette, Rosenfeld, Ron G, Yeste Fernández, Diego, Yorifuji, Tohru, Blum, Werner F, and Universitat Autònoma de Barcelona

Subjects
Male, medicine.medical_specialty, Pediatrics, Diabetes risk, Adolescent, Endocrinology, Diabetes and Metabolism, Clinical Biochemistry, Population, 030209 endocrinology & metabolism, Biochemistry, Short stature, 03 medical and health sciences, 0302 clinical medicine, Endocrinology, Risk Factors, Neoplasms, Internal medicine, Turner syndrome, medicine, Humans, Prospective Studies, Child, education, Prospective cohort study, Growth Disorders, Clinical Research Articles, Cerebral Hemorrhage, education.field_of_study, Human Growth Hormone, business.industry, Incidence, Incidence (epidemiology), Biochemistry (medical), medicine.disease, Recombinant Proteins, 3. Good health, Idiopathic short stature, Growth, Growth Hormone, and Growth Factors, Standardized mortality ratio, Diabetes Mellitus, Type 2, Child, Preschool, Female, medicine.symptom, business, 030217 neurology & neurosurgery, Follow-Up Studies
Abstract

Context Safety concerns have been raised regarding premature mortality, diabetes, neoplasia, and cerebrovascular disease in association with GH therapy. Objective To assess incidence of key safety outcomes. Design Prospective, multinational, observational study (1999 to 2015). Setting A total of 22,311 GH-treated children from 827 investigative sites in 30 countries. Patients Children with growth disorders. Interventions GH treatment. Main outcome measures Standardized mortality ratio (SMR) and standardized incidence ratio (SIR) with 95% CIs for mortality, diabetes, and primary cancer using general population registries. Results Predominant short stature diagnoses were GH deficiency (63%), idiopathic short stature (13%), and Turner syndrome (8%), with mean ± SD follow-up of 4.2 ± 3.2 years (∼92,000 person-years [PY]). Forty-two deaths occurred in patients with follow-up, with an SMR (95% CI) of 0.61 (0.44, 0.82); the SMR was elevated for patients with cancer-related organic GH deficiency [5.87 (3.21, 9.85)]. Based on 18 cases, type 2 diabetes mellitus (T2DM) risk was elevated [SIR: 3.77 (2.24, 5.96)], but 72% had risk factors. In patients without cancer history, 14 primary cancers were observed [SIR: 0.71 (0.39, 1.20)]. Second neoplasms occurred in 31 of 622 cancer survivors [5.0%; 10.7 (7.5, 15.2) cases/1000 PY] and intracranial tumor recurrences in 67 of 823 tumor survivors [8.1%; 16.9 (13.3, 21.5) cases/1000 PY]. All three hemorrhagic stroke cases had risk factors. Conclusions GeNeSIS (Genetics and Neuroendocrinology of Short Stature International Study) data support the favorable safety profile of pediatric GH treatment. Overall risk of death or primary cancer was not elevated in GH-treated children, and no hemorrhagic strokes occurred in patients without risk factors. T2DM incidence was elevated compared with the general population, but most cases had diabetes risk factors., Safety of GH therapy was assessed in a pediatric observational study. Death and primary cancer rates were not higher than in the general population; T2DM rate was higher owing to risk factors.

Published
2018
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24. 129-LB: Evaluation of Attitudes towards Two Glucagon Delivery Devices for Rescue Treatment of Severe Hypoglycemia

Author

BAJPAI, SANJAY K., primary, CAMBRON-MELLOTT, JANELLE, additional, RAIBULET, NEDINA K., additional, PECK, EUGENIA Y., additional, POON, JIAT LING, additional, WANG, QIANQIAN, additional, MITCHELL, BETH, additional, WILL, OLIVER, additional, BABROWICZ, JANE, additional, CHILD, CHRISTOPHER J., additional, and BEUSTERIEN, KATHLEEN, additional

Published
2020
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28. Structural similarities between 6-methylsalicylic acid synthase from Penicillium patulum and vertebrate type I fatty acid synthase: evidence from thiol modification studies

Author

Child, Christopher J., Spencer, Jonathan B., Bhogal, Pamela, and Shoolingin-Jordan, Peter M.

Subjects
Enzymes -- Research, Biosynthesis -- Research, Biological sciences, Chemistry
Abstract

The multifunctional enzyme complex from Penicillium patulum called 6-methylsalicylic acid synthase, that catalyzes the biosynthesis of the tetraketide 6-methylsalicylic acid, was altered by thiol-specific inhibitors and cross-linking reagents. Treatment with 1,3-dibromopropan-2-one caused rapid enzyme inactivation and formation of cross-linked dimers. Two types of cross-linked dimers were formed. Within each dimer, the beta-ketoacyl synthase and acyl carrier protein components are juxtaposed to enable the respective cysteine and 4'-phosphopantetheine thiols to interact during condensation.

Published
1996

31. 285-OR: Conversations and Reactions around Severe Hypoglycemia (CRASH): U.S. Results from a Global Survey of People with T1DM or Insulin-Treated T2DM and Caregivers

Author

SNOEK, FRANK J., primary, JILETCOVICI, ALINA, additional, BUSHNELL, DONALD M., additional, CHILD, CHRISTOPHER J., additional, BAJPAI, SANJAY K., additional, SPAEPEN, ERIK, additional, THOMAS, JOSH, additional, BALANTAC, ZANETA, additional, PEYROT, MARK, additional, and MITCHELL, BETH, additional

Published
2019
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34. Effect of growth hormone（GH）treatment on adult height in patients with GH deficiency：the Japanese cohort from the GeNeSIS observational study.

Author

Toshiaki Tanaka, Tomonobu Hasegawa, Keiichi Ozono, Hiroyuki Tanaka, Susumu Kanzaki, Susumu Yokoya, Kazuo Chihara, Tomonori Oura, Katsuichiro Ihara, Child, Christopher J., and Yoshiki Seino

Subjects
SOMATOTROPIN, PITUITARY dwarfism, NEUROENDOCRINOLOGY, DESCRIPTIVE statistics, SCIENTIFIC observation
Abstract

This study evaluated the effect of growth hormone (GH) treatment on adult height in pediatric patients with growth hormone deficiency (GHD) using data from the Japanese cohort of the multinational observational Genetics and Neuroendocrinology of Short Stature International Study (GeNeSIS, NCT01088412). We assessed pediatric patients with GHD who received GH therapy and attained adult height, defined as chronological age ≥12 years in girls and ≥14 years in boys and ≥1 of the following: closed epiphyses, height velocity ＜2 cm/year, or a bone age ≥14 years in girls and ≥16 years in boys. Descriptive statistics for endpoints by gender were calculated using the standards in 2000 for growth chart in Japan. Subgroup analyses were conducted for starting age of GH treatment, concomitant therapy, and mean GH treatment dose. Of the 2001 Japanese children with GHD, there were 230（boys＝130；girls＝100）who achieved adult height during the study. The mean（±standard deviation [SD])adult height was 159.6±5.4 cm in boys and 146.6±5.7 cm in girls. The change in height standard deviation score was 0.92±0.85 and 0.99±0.91 in boys and girls, respectively. In the subgroup analysis by concomitant therapy, a significant difference（p＜0.05）was observed in adult height between boys with and without anabolic steroid hormone use (off—label use）. An analysis of adult height by GH treatment dose and by age showed no statistically significant differences. In conclusion, GH—treated Japanese children with GHD exhibited clear improvement in adult height gain. [ABSTRACT FROM AUTHOR]

Published
2020

35. Screening a large pediatric cohort with GH deficiency for mutations in genes regulating pituitary development and GH secretion: Frequencies, phenotypes and growth outcomes

Author

Blum, Werner F., primary, Klammt, Jürgen, additional, Amselem, Serge, additional, Pfäffle, Heike M., additional, Legendre, Marie, additional, Sobrier, Marie-Laure, additional, Luton, Marie-Pierre, additional, Child, Christopher J., additional, Jones, Christine, additional, Zimmermann, Alan G., additional, Quigley, Charmian A., additional, Cutler, Gordon B., additional, Deal, Cheri L., additional, Lebl, Jan, additional, Rosenfeld, Ron G., additional, Parks, John S., additional, and Pfäffle, Roland W., additional

Published
2018
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37. Growth Hormone Treatment for Short Stature in the USA, Germany and France: 15 Years of Surveillance in the Genetics and Neuroendocrinology of Short-Stature International Study (GeNeSIS)

Author

Pfaeffle, Roland, Land, Christof, Schoenau, Eckhard, Holterhus, Paul-Martin, Ross, Judith L., de Oliveira, Carolina Piras, Child, Christopher J., Benabbad, Imane, Jia, Nan, Jung, Heike, Blum, Werner F., Pfaeffle, Roland, Land, Christof, Schoenau, Eckhard, Holterhus, Paul-Martin, Ross, Judith L., de Oliveira, Carolina Piras, Child, Christopher J., Benabbad, Imane, Jia, Nan, Jung, Heike, and Blum, Werner F.

Abstract

Background/Aims: To describe characteristics, auxological outcomes and safety in paediatric patients with growth disorders treated with growth hormone (GH), for cohorts from the USA, Germany and France enrolled in GeNeSIS, a postauthorisation surveillance programme. Methods: Diagnosis and biochemical measurement data were based on reporting from, and GH treatment was initiated at the discretion of, treating physicians. Auxological outcomes during the first 4 years of GH treatment and at near-adult height (NAH) were analysed. Serious and treatment-emergent adverse events were described. Results: Children in the USA (n = 9,810), Germany (n = 2,682) and France (n = 1,667) received GH (dose varied between countries), most commonly for GH deficiency. Across diagnostic groups and countries, mean height velocity standard deviation score (SDS) was > 0 and height SDS increased from baseline during the first 4 years of treatment, with greatest improvements during year 1. Most children achieved NAH within the normal range (height SDS >-2). No new or unexpected safety concerns were noted. Conclusion: GH treatment improved growth indices to a similar extent for patients in all three countries despite variations in GH doses. Data from these three countries, which together contributed > 60% of patients to GeNeSIS, indicated no new safety signals and the benefit-risk profile of GH remains unchanged. (c) 2018 S. Karger AG, Basel

Published
2018

38. Responses to the Letter to the Editor “Does growth-hormone treatment affect patients with and without a mitochondrial disorder differentially ?” (Vol. 27, No. 2, p. 107–108, 2018)

Author

Yokoya, Susumu, primary, Hasegawa, Tomonobu, additional, Ozono, Keiichi, additional, Tanaka, Hiroyuki, additional, Kanzaki, Susumu, additional, Tanaka, Toshiaki, additional, Chihara, Kazuo, additional, Jia, Nan, additional, Child, Christopher J., additional, Ihara, Katsuichiro, additional, Funai, Jumpei, additional, Iwamoto, Noriyuki, additional, and Seino, Yoshiki, additional

Published
2018
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39. Growth Hormone Treatment for Short Stature in the USA, Germany and France: 15 Years of Surveillance in the Genetics and Neuroendocrinology of Short-Stature International Study (GeNeSIS)

Author

Pfäffle, Roland, primary, Land, Christof, additional, Schönau, Eckhard, additional, Holterhus, Paul-Martin, additional, Ross, Judith L., additional, Piras de Oliveira, Carolina, additional, Child, Christopher J., additional, Benabbad, Imane, additional, Jia, Nan, additional, Jung, Heike, additional, and Blum, Werner F., additional

Published
2018
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40. THE GENETICS AND NEUROENDOCRINOLOGY OF SHORTSTATURE INTERNATIONAL STUDY (GENESIS): DATA FROM 15 YEARS OF SURVEILLANCE OF GROWTH HORMONE THERAPY IN GERMANY, FRANCE AND THE USA

Author

Pfaeffle, Roland, Land, Christof, Schoenau, Eckhard, Holterhus, Paul Martin, Ross, Judith, De Oliveira, Carolina Piras, Child, Christopher J., Benabbad, Imane, Jia, Nan, Jung, Heike, Blum, Werner F., Pfaeffle, Roland, Land, Christof, Schoenau, Eckhard, Holterhus, Paul Martin, Ross, Judith, De Oliveira, Carolina Piras, Child, Christopher J., Benabbad, Imane, Jia, Nan, Jung, Heike, and Blum, Werner F.

Published
2017

41. Incidence of diabetes mellitus and neoplasia in Japanese short-statured children treated with growth hormone in the Genetics and Neuroendocrinology of Short Stature International Study (GeNeSIS)

Author

Yokoya, Susumu, primary, Hasegawa, Tomonobu, additional, Ozono, Keiichi, additional, Tanaka, Hiroyuki, additional, Kanzaki, Susumu, additional, Tanaka, Toshiaki, additional, Chihara, Kazuo, additional, Jia, Nan, additional, Child, Christopher J., additional, Ihara, Katsuichiro, additional, Funai, Jumpei, additional, Iwamoto, Noriyuki, additional, and Seino, Yoshiki, additional

Published
2017
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42. Safety Outcomes and Near-Adult Height Gain of Growth Hormone-Treated Children with SHOX Deficiency: Data from an Observational Study and a Clinical Trial

Author

Benabbad, Imane, primary, Rosilio, Myriam, additional, Child, Christopher J., additional, Carel, Jean-Claude, additional, Ross, Judith L., additional, Deal, Cheri L., additional, Drop, Stenvert L.S., additional, Zimmermann, Alan G., additional, Jia, Nan, additional, Quigley, Charmian A., additional, and Blum, Werner F., additional

Published
2016
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44. Safety Outcomes During Pediatric GH Therapy: Final Results From the Prospective GeNeSIS Observational Program.

Author

Child, Christopher J, Zimmermann, Alan G, Chrousos, George P, Cummings, Elisabeth, Deal, Cheri L, Hasegawa, Tomonobu, Jia, Nan, Lawrence, Sarah, Linglart, Agnès, Loche, Sandro, Maghnie, Mohamad, Pérez Sánchez, Jacobo, Polak, Michel, Predieri, Barbara, Richter-Unruh, Annette, Rosenfeld, Ron G, Yeste, Diego, Yorifuji, Tohru, and Blum, Werner F

Abstract

Safety concerns have been raised regarding premature mortality, diabetes, neoplasia, and cerebrovascular disease in association with GH therapy.

Published
2019
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47. Incidence of diabetes mellitus and neoplasia in Japanese short-statured children treated with growth hormone in the Genetics and Neuroendocrinology of Short Stature International Study (GeNeSIS).

Author

Susumu Yokoya, Tomonobu Hasegawa, Keiichi Ozono, Hiroyuki Tanaka, Susumu Kanzaki, Toshiaki Tanaka, Kazuo Chihara, Jia, Nan, Child, Christopher J., Katsuichiro Ihara, Jumpei Funai, Noriyuki Iwamoto, and Yoshiki Seino

Subjects
DIABETES, LACTIC acidosis, CANCER, INSULIN, ENCEPHALOMYELITIS
Abstract

The primary goal of the Genetics and Neuroendocrinology of Short Stature International Study (GeNeSIS) was to assess the safety and effectiveness of Humatrope®, a GH preparation, in the treatment of pediatric patients with short stature. We report our findings in the GH-treated Japanese pediatric population focusing on the incidence of type 2 diabetes (T2D) and occurrence of neoplasms. A total of 2,345 Japanese patients were assessed for safety. During a mean observation period of 3.2 yr, T2D occurred in 3 patients (0.13%) and slowly progressive insulin-dependent diabetes mellitus (SPIDDM) related to underlying mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS) in 1 patient (0.04%). Neoplasms were reported in 13 patients (0.56%), including 1 patient with brain tumor (germinoma) and 5 with craniopharyngiomas (4 recurrences); the remainder were benign, typically dermatological, neoplasms. The incidence of diabetes mellitus determined in the study did not differ from previous reports in GH-treated pediatric patients, and there was no apparent increase in the risk of new neoplastic lesions or malignant tumors. [ABSTRACT FROM AUTHOR]

Published
2017
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48. Safety Outcomes and Near-Adult Height Gain of Growth Hormone-Treated Children with SHOX Deficiency: Data from an Observational Study and a Clinical Trial.

Author

Benabbad, Imane, Rosilio, Myriam, Child, Christopher J., Carel, Jean-Claude, Ross, Judith L., Deal, Cheri L., Drop, Stenvert L.S., Zimmermann, alan G., Jia, Nan, Quigley, Charmian a., and Blum, Werner F.

Subjects
SOMATOTROPIN, CONFIDENCE intervals, STANDARD deviations
Abstract

Background/Aims: To assess auxological and safety data for growth hormone (GH)-treated children with SHOX deficiency. Methods: Data were examined for GH-treated SHOX-deficient children ( n = 521) from the observational Genetics and Neuroendocrinology of Short Stature International Study (GeNeSIS). For patients with near-adult height information, GeNeSIS results ( n = 90) were compared with a clinical trial ( n = 28) of SHOX-deficient patients. Near-adult height was expressed as standard deviation score (SDS) for chronological age, potentially increasing the observed effect of treatment. Results: Most SHOX-deficient patients in GeNeSIS had diagnoses of Leri-Weill syndrome ( n = 292) or non-syndromic short stature ( n = 228). For GeNeSIS patients with near-adult height data, mean age at GH treatment start was 11.0 years, treatment duration 4.4 years, and height SDS gain 0.83 (95% confidence interval 0.49-1.17). Respective ages, GH treatment durations and height SDS gains for GeNeSIS patients prepubertal at baseline ( n = 42) were 9.2 years, 6.0 years and 1.19 (0.76-1.62), and for the clinical trial cohort they were 9.2 years, 6.0 years and 1.25 (0.92-1.58). No new GH-related safety concerns were identified. Conclusion: Patients with SHOX deficiency who had started GH treatment before puberty in routine clinical practice had a similar height gain to that of patients in the clinical trial on which approval for the indication was based, with no new safety concerns. [ABSTRACT FROM AUTHOR]

Published
2017
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49. Radiological Features in Patients with Short Stature Homeobox-Containing (SHOX) Gene Deficiency and Turner Syndrome before and after 2 Years of GH Treatment

Author

Child, Christopher J., primary, Kalifa, Gabriel, additional, Jones, Christine, additional, Ross, Judith L., additional, Rappold, Gudrun A., additional, Quigley, Charmian A., additional, Zimmermann, Alan G., additional, Garding, Gina, additional, Cutler Jr., Gordon B., additional, and Blum, Werner F., additional

Published
2015
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