Publications by authors named "Cecilia Camacho-Hubner"
Objectives: To identify and describe the profile of potential transthyretin cardiac amyloidosis (ATTR-CM) cases in the Brazilian public health system (SUS), using a predictive machine learning (ML) model.
Methods: This was a retrospective descriptive database study that aimed to estimate the frequency of potential ATTR-CM cases in the Brazilian public health system using a supervised ML model, from January 2015 to December 2021. To build the model, a list of ICD-10 codes and procedures potentially related with ATTR-CM was created based on literature review and validated by experts.
Article Synopsis
- The study aimed to evaluate the safety and effectiveness of growth hormone (GH) replacement therapy in adults with Langerhans cell histiocytosis (LCH) who also have growth hormone deficiency (GHD), using data from the Pfizer International Metabolic Database (KIMS).
- Out of 81 patients analyzed, one year of GH treatment showed improvements in metabolic parameters like reduced total cholesterol and increased quality of life scores, with 37 patients providing follow-up data.
- The safety analysis revealed 36 serious adverse events over 435 patient-years, but no new safety concerns were identified, suggesting that GH treatment is safe and effective for this population.
Objective: To report the final long-term safety and efficacy analyses of patients with acromegaly treated with pegvisomant from the ACROSTUDY.
Design: Global (15 countries), multicentre, non-interventional study (2004-2017).
Methods: The complete ACROSTUDY cohort comprised patients with acromegaly, who were being treated with pegvisomant (PEGV) prior to the study or at enrolment.
Article Synopsis
- Cancer survivors with growth hormone deficiency (GHD) receive growth hormone therapy (GHT) after being monitored for at least a year to ensure their tumor status is stable.
- The study investigates how different types and locations of radiation therapy (RT) impact patients' growth responses to GHT.
- Findings reveal that leukemia patients who underwent total body irradiation (TBI) experienced the least growth response, while those with craniopharyngioma showed the best growth response, indicating varying effects of RT on GHT efficacy.
A Genome-Wide Pharmacogenetic Study of Growth Hormone Responsiveness.
J Clin Endocrinol Metab
October 2020
Context: Individual patients vary in their response to growth hormone (GH). No large-scale genome-wide studies have looked for genetic predictors of GH responsiveness.
Objective: To identify genetic variants associated with GH responsiveness.
Article Synopsis
- Children born prematurely and treated with growth hormone (GH) showed a significant increase in height during the early years of treatment.
- A study of 586 preterm children analyzed the effects of GH on near-adult height (NAH), revealing that those born appropriate for gestational age (AGA) had a better growth response than those born small for gestational age (SGA).
- The findings suggest that GH treatment is beneficial, especially in prepubertal children, and that parental height is a strong predictor of growth outcomes, with prematurity not impacting the overall growth response.
Article Synopsis
- The study examines how accurately adult height can be predicted in children with idiopathic growth hormone deficiency (IGHD) using bone age methods, specifically the Bayley-Pinneau (BP) and Tanner-Whitehouse 2 (TW2) methods.
- Results show that both prediction methods initially underestimate adult height at the start of growth hormone (GH) treatment, but after several years of treatment, they tend to overestimate adult height.
- The findings suggest that adjustments in height predictions are necessary for better accuracy in children with IGHD throughout their treatment journey.
Objective: Clinical observations over time of adults with growth hormone (GH) deficiency (GHD) have indicated a shift in patient characteristics at diagnosis. The objective of this study was to compare baseline characteristics of patients diagnosed with adult-onset GHD naive to GH replacement during three study periods (1994-1999 (P1), 2000-2004 (P2), and 2005-2012 (P3)) using the KIMS (Pfizer's International Metabolic) database.
Methods: Data were retrieved for a total of 6069 patients with adult-onset GHD from six countries (Belgium, Germany, Netherlands, Spain, Sweden, and UK): P1 (n = 1705), P2 (n = 2397), and P3 (n = 1967).
Article Synopsis
- The study aimed to investigate how spontaneous versus induced puberty and the type of estrogen treatment affect the height of girls with Turner syndrome undergoing growth hormone (GH) therapy.
- Researchers analyzed data from 772 girls, comparing those who experienced spontaneous puberty (145 girls) with those who required estrogen induction (627 girls).
- The findings indicated that girls with spontaneous puberty showed slightly better growth in response to GH, leading to earlier adult height, but overall height differences between the two groups were not significant at maturity.
Background/aims: There is little information how rhGH treatment affects height in NS. This study aims to analyze data from the NS patients assembled in KIGS over 25 years.
Patients/methods: Of 613 (389 m/224 f) NS patients documented, 476 (302 m/174 f) were treated for 1 year, 237 (160 m/77 f) of which served to develop a 1st year height velocity (HV) prediction algorithm.
Background/aims: It is unknown whether long-term growth hormone replacement therapy (GHRT) affects body composition in an age- or sex-dependent manner. We aimed to study the effects of 4 years of GHRT on body composition in a large cohort of patients with hypopituitarism compared to a reference population matched by age and sex.
Methods: A total of 964 GH-deficient adults from KIMS (Pfizer International Metabolic Database) with adult-onset hypopituitarism, adequately replaced with all pituitary hormones except for GH at baseline were included.
Purpose: To explore the effects of pegvisomant (PEGV) on glucose metabolism in patients with acromegaly within ACROSTUDY, an international, observational, prospective safety surveillance study.
Methods: Patients were retrospectively divided into two cohorts, with (DM group) or without diabetes mellitus (no-DM). Parameters of glucose metabolism and IGF-I values were analyzed yearly both cross-sectionally for 4 years (yrs) and longitudinally at 1 and 4-5 yrs of PEGV treatment.
Background: Quality of life (QoL) and health economic data are becoming increasingly important factors in healthcare decision making. While there is a wealth of information establishing the benefit of growth hormone (GH) replacement therapy in adults with growth hormone deficiency (aGHD), recent reviews on the QoL and health economic impact of aGHD and the effect of treatment on these factors is limited.
Objective: The aim of this article is to summarize the impact of early and sustained treatment on the QoL and economic burden of aGHD by conducting a targeted literature review.
Objectives ACROSTUDY is an international, non-interventional study of acromegaly patients treated with pegvisomant (PEGV), a growth hormone receptor antagonist and has been conducted since 2004 in 15 countries to study the long-term safety and efficacy of PEGV. This report comprises the second interim analysis of 2090 patients as of May 12, 2016. Methods Descriptive analyses of safety, pituitary imaging and outcomes on PEGV treatment up to 12 years were performed.
View Article and Find Full Text PDFBackground/aims: To compare racial/ethnic proportions of subjects receiving growth hormone (GH) treatment to the expected proportions, and secondarily, to assess racial/ethnic differences in subject characteristics at GH treatment initiation.
Methods: Race/ethnicity-based expected frequencies of height <-2.25 SD were determined by applying relative risks for short stature, calculated from a regional population of 189,280 pediatric primary care patients, to US census data, and compared to racial/ethnic proportions of US subjects enrolled in the Pfizer International Growth Study (KIGS) using the χ2 test.
Background: Adult-onset growth hormone deficiency (AO-GHD) is associated with an increased prevalence of the metabolic syndrome (MetS).
Aim: To determine the effect of GH replacement on the prevalence of MetS in AO-GHD and to study the impact of MetS on the incidence of cardiovascular events during GH replacement.
Patients And Methods: 1449 AO-GHD patients (males 48.
Article Synopsis
- The objective of the study was to analyze the growth response and GH dosage in prepubertal patients who have both type 1 diabetes mellitus (T1DM) and growth hormone deficiency (GHD).
- The study included 69 patients with T1DM and GHD, and their growth metrics were compared to a control group of prepubertal patients with GHD only, revealing no significant differences in age, height, or GH dosage.
- The findings indicated that the first-year growth response to GH therapy was similar for both groups, suggesting that T1DM does not negatively affect GH treatment outcomes, supporting its safety for these patients.
Secular Trends on Birth Parameters, Growth, and Pubertal Timing in Girls with Turner Syndrome.
Front Endocrinol (Lausanne)
February 2018
Background: Whether children with chromosomal disorders of growth and puberty are affected by secular trends (STs) as observed in the general population remains unanswered, but this question has relevance for expectations of spontaneous development and treatment responses.
Objectives: The aim of the study was to evaluate STs in birth parameters, growth, and pubertal development in girls with Turner syndrome (TS).
Study Design: Retrospective analysis of KIGS data (Pfizer International Growth Database).
Context: Data on the association between growth hormone (GH) replacement in patients with GH deficiency (GHD) after malignancies and new neoplasms show conflicting results.
Objective: To clarify the incidence of new malignant neoplasm in childhood-onset (CO) and adult-onset (AO) adult cancer survivors (CSs).
Design: Retrospective comparison of CO-CS and AO-CS with CO idiopathic GHD (IGHD) and AO nonfunctioning pituitary adenoma (NFPA) patients and with the general population [standardized incidence ratio (SIR)].
Context: In adults, craniopharyngioma (CP) of either childhood-onset (CO-CP) or adult-onset (AO-CP) is associated with increased morbidity and mortality, but data on the relative risks (RRs) of contributing factors are lacking.
Objective: To assess the RRs of factors contributing to morbidity and mortality in adults with CO-CP and AO-CP.
Methods: Data on 1669 patients with CP from KIMS (Pfizer International Metabolic Database) were analyzed using univariate and multiple Poisson and Cox regression methods.
Background: Growth prediction models (GPMs) exist to support clinical management of children treated with growth hormone (GH) for growth hormone deficiency (GHD), Turner syndrome (TS) and for short children born small for gestational age (SGA). Currently, no prediction system has been widely adopted.
Content: The objective was to develop a stand-alone web-based system to enable the widespread use of an 'individualised growth response optimisation' (iGRO) tool across European endocrinology clinics.
Context: De novo brain tumors developing after treatment of pituitary/sellar lesions have been reported, but it is unknown whether this is linked to any of the treatment modalities.
Objective: To study the occurrence of malignant brain tumors and meningiomas in a large cohort of patients treated for pituitary/sellar lesions, with special emphasis on the role of radiotherapy (RT).
Patients And Methods: Patients (n = 8917) who were hypopituitary due to pituitary adenomas, craniopharyngiomas, and other sellar tumors followed in KIMS (Pfizer International Metabolic Database) from 1994 to 2012 were included.
Context: Longitudinal data of children with Prader-Willi syndrome (PWS) treated with genotropin were registered in the Pfizer International Growth Database (KIGS).
Objective: To evaluate efficacy and safety of growth hormone (GH) treatment in a large group of children with PWS.
Design: Data registered in KIGS from 1987 to 2012.
Background: Information about disease-specific and gender-associated differences over longer time of short children treated with recombinant human growth hormone is missing.
Methods: We analyzed data at growth hormone (GH) start in prepubertal children diagnosed with idiopathic GH deficiency (IGHD), congenital GHD, acquired GHD, idiopathic short stature (ISS), and born small for gestational age (SGA) enrolled (1987-2012) in the Pfizer International Growth Study (KIGS®) from Europe, USA, and Japan.
Results: The demographic characteristics of patients in the three regions were similar.