Growth hormone replacement in adults: Real-world data from two large studies in US and Europe.

OBJECTIVE: This report describes the effectiveness and safety of growth hormone replacement in 3180 adult patients with growth hormone deficiency followed-up for 0.0-12.2 years in two completed, complementary, non-interventional, multicentre studies, NordiNet® International Outcome Study (IOS) (NCT00960128) and the American Norditropin® Studies: Web-Enabled Research (ANSWER) Program (NCT01009905). DESIGN: In both studies, Norditropin® (somatropin; Novo Nordisk A/S, Denmark) was administered at the discretion of the treating physician and according to routine practice. We present data on baseline characteristics, growth hormone dose, safety data and change from baseline in waist circumference, body mass index and bioimpedance (NordiNet® IOS only). RESULTS: Mean (SD) baseline characteristics (effectiveness analysis set) in NordiNet® IOS (n = 971) and ANSWER (n = 304): females, 45%; 69%; mean growth hormone dose (mg/day) (female, 0.338 [0.177]; male, 0.289 [0.157]); (female, 0.501 [0.313]; male, 0.505 [0.351]). Most patients had BMI ≥25 kg/m2. Median (P10,P90) exposure (females, 3.5 [0.42,11.0]; 1.6 [3.2; 0.3,8.6]; males, 4.1 [0.33,10.8]; 2.3 [2.9; 0.0,7.5] years). Mean (SD) change from baseline for waist circumference (-0.46 [6.38] cm [n = 403], BMI (0.30 [3.30] kg/m2 [n = 857]) and bioimpedance (-17.4 (59.19) ohm [n = 239]) were associated with growth hormone dose (waist/bioimpedance) and duration of follow-up (BMI/bioimpedance). No new safety signals were observed among patients in the full analysis set (NordiNet® IOS, n = 2321; ANSWER, n = 859). CONCLUSIONS: Long-term growth hormone replacement is associated with an improvement in body composition. The accumulated data from >10 years of follow-up support the long-term effectiveness and safety of growth hormone replacement as prescribed in clinical practice.

Educational needs of hematologists and laboratory professionals regarding factor activity assays.

INTRODUCTION: Diagnosis and management of hemophilia require accurate and precise measurements of factor activity levels. Activity is traditionally measured via one-stage (OS) clot-based assay; however, chromogenic substrate (CS) assays may be needed for certain cases. A survey was performed to understand assay-related knowledge gaps among hematologists and laboratory professionals. METHODS: Separate web-based surveys were administered to hematologists who manage hemophilia and to laboratory professionals and queried practice patterns, knowledge of/attitudes toward CS assays, and interest in continuing education. RESULTS: A total of 51 hematologists participated in this study; 67% managed hemophilia patients for ≥10 years and 24% were affiliated with a hemophilia treatment center (HTC). Most (80%) stated familiarity with general assay interpretation. Majorities of non-HTC and HTC respondents agreed that CS assays are more accurate than OS assays (62%/67%), although non-HTC hematologists indicated less understanding of when to order a CS assay (49%/67%). Fewer non-HTC respondents expressed concerns regarding the reliability of OS assays for diagnosis (38%/67%) and monitoring (38%/75%). Most (80%) expressed an interest in factor assay education, especially on available assays, efficacy, and best practices (39%). A total of 57 laboratory professionals participated, averaging 10 years in their current position; most (88%) were hospital based. More performed OS (72%) than CS (10%) or both (17%) assays; only 11% reported confidence with the interpretation of CS results. Few expressed concerns regarding the reliability of OS for diagnosis (9%) or monitoring (12%). Reported barriers to CS use included infrequent need (68%), lack of US Food and Drug Administration (FDA) approval (61%), and need for validation work (56%). Most (70%) were interested in CS assay education; top interests included advantages over traditional assays, general information on CS assays, and indications for testing (each 18%). CONCLUSION: Future educational efforts may focus on limitations of OS assays, indications for CS assay diagnosis/monitoring, and support for clinic-laboratory dialog.

The Coags Uncomplicated App: Fulfilling Educational Gaps Around Diagnosis and Laboratory Testing of Coagulation Disorders.

BACKGROUND: Patients with coagulation disorders may present to a variety of physician specialties; however, accurate and efficient diagnosis can be challenging for physicians not specialized in hematology, due to identified gaps in knowledge around appropriate laboratory assays and interpretation of test results. Coags Uncomplicated was developed to fill this unmet educational need by increasing practical knowledge of coagulation disorders among nonexpert physicians and other health care professionals (HCPs) in a point-of-care (POC) setting. OBJECTIVE: The aim of this study was to assess patterns of use of the mobile app Coags Uncomplicated, a tool designed to support education regarding accurate and efficient diagnosis of bleeding disorders. METHODS: App metrics were obtained by tracking registered user data. Additionally, a survey was distributed to registered users, to assess circumstances and frequency of use. RESULTS: The most common specialties of 7596 registered US users were hematology-oncology (n=1534, 20.19%), hematology (n=1014, 13.35%), and emergency medicine (n=1222, 16.09%); most identified as physicians (n=4082, 53.74%). Specialties accounting for the greatest numbers of screen views were hematology-oncology (99,390 views), hematology (47,808 views), emergency medicine (23,121 views), and internal medicine (22,586 views). The most common diagnostic endpoints reached were disseminated intravascular coagulation (DIC; 2713 times), liver disease effect (2108 times), and vitamin K deficiency (1584 times). Of 3424 users asked to take the survey, 262 responded (7.65%); most were physicians in direct clinical care (71%) and specialized in hematology-oncology (39%) or emergency medicine (21%). Most frequent use was reported by hematologists (69%, ≥6 times) and hematologists-oncologists (38%, ≥6 times). Most physicians (89.2%) reported using the app for patient-case-related education around appropriate use of laboratory tests in diagnostic evaluation. Physicians rated Lab Value Analyzer (mean 4.43) and Lab Test Algorithm (mean 4.46) tools highly on a 5-point "how helpful" scale and were likely to recommend the app to colleagues. CONCLUSIONS: App use among physicians and other HCPs is consistent with value as a POC educational tool, which may facilitate differential diagnoses and appropriate early consultation with hematologists.