Resumen es: El miocardio no compactado es una entidad de origen congenito que pertenece al grupo de las miocardiopatias no clasificables. Su asociacion con la esf...
Los tumores cardíacos primarios constituyen una entidad rara. Sólo el 10% a 25% de ellos son malignos y el angiosarcoma es el más frecuente. Ocasionan sintomatología cardíaca y sistémica variada, por sí mismos o por compromiso de otros órganos, lo que puede simular otras patologías y derivar en un retraso del diagnóstico de certeza. Se describen dos casos de angiosarcoma cardíaco con presentaciones atípicas, por lo que inicialmente no se consideró esta patología como un diagnóstico probable. Se requiere un alto grado de sospecha clínica, junto con métodos por imágenes de alta resolución como la resonancia magnética, para la aproximación diagnóstica correcta y el tratamiento adecuado.
Background Physical function assessment is key for the management of knee musculoskeletal conditions. There are a wide variety of self-reported outcome measures (SROMs) and performance-based outcome measures (PBOMs) to assess physical function of individuals with knee conditions. However, the content of these measures has not been explored. Objective To explore the range and frequency of physical functions assessed by lower limb PBOMs and SROMs for people with knee osteoarthritis (OA), anterior cruciate ligament (ACL) injuries, and patellofemoral pain (PFP). Methods A scoping review was conducted. We included development or measurement properties studies of knee functional outcome measures for populations with knee OA, ACL injuries, and PFP. We extracted the physical functions assessed in each measure. Each identified physical function was linked to a code from the International Classification of Functioning, Disability and Health (ICF) framework. Results 4146 articles were screened. A total of 143 articles were included. The median number of physical functions assessed was nine for SROMs and one for PBOMs. The three most assessed physical functions were climbing stairs, walking short distances, and standing up from sitting. Climbing stairs was the most assessed physical function in measures for knee OA and PFP populations, whereas jumping was in measures for the ACL-injured population. Conclusion SROMs assess a broader range of physical functions, whereas PBOMs focus on discrete activities. ACL and PFP measures evaluated more challenging physical functions than knee OA measures. Current physical function outcome measures are not well suited to assess performance in knee OA populations with mild or diverse levels of impairment.
Disclosure: B.M. Biller: Crinetics Pharmaceuticals, Inc., Principal investigator of a research grant to Massachusetts General Hospital, consultant, Amolyt Pharma, Consultant, Camurus, Consultant, Chiesi, Consultant, Pfizer, Consultant, Recordati, Consultant. A. Casagrande: Crinetics Pharmaceuticals, Employee and shareholder. C.J. Strasburger: Crinetics Pharmaceuticals, Principal investigator of a research grant, consultant/speaker, Amolyt Pharma, Consultant/speaker, Novo Nordisk, Consultant/speaker, Pfizer, Consultant/speaker, Recordati, Consultant/speaker, Sandoz-Hexal, Consultant/speaker. M. Bidlingmaier: Amolyt Pharma, Principal investigator of a research grant, Camurus, Principal investigator of a research grant, Chiasma, Principal investigator of a research grant, Crinetics Pharmaceuticals, Inc., Principal investigator of a research grant, consultant, IDS, Principal investigator of a research grant, Ionis, Principal investigator of a research grant, consultant, Lumos, Principal investigator of a research grant, OPKO, Principal investigator of a research grant, Novo Nordisk, Consultant and speaker, Pfizer, Consultant and speaker, Roche, Consultant, Sandoz, Consultant, Euroimmun, Speaker. P.J. Snyder: Crinetics Pharmaceuticals, Inc., Principal investigator of a research grant. M.A. Guitelman: Crinetics Pharmaceuticals, Inc., Principal investigator of a research grant, Recordati, Consultant/speaker, Sanofi Aventis, Consultant/speaker. C.L. Boguszewski: Crinetics Pharmaceuticals, Inc., Principal investigator of a research grant, Ipsen, Consultant/speaker, Recordati, Consultant/speaker. M. Buchfelder: Crinetics Pharmaceuticals, Inc., Principal investigator of a research grant, Novo Nordisk, Speaker. I. Shimon: Crinetics Pharmaceuticals, Inc., Principal investigator of a research grant, Debiopharm, Principal investigator of a research grant, Medison, Consultant/speaker, Opko, Consultant/speaker, Pfizer, Consultant/speaker. G. Raverot: Crinetics Pharmaceuticals, Inc., Principal investigator of a research grant pid to institution, Novo Nordisk, Consultant/speaker, Pfizer, Consultant/speaker, Recordati, Consultant/speaker. M. Toth: Crinetics Pharmaceuticals, Inc., Principal investigator of a research grant, Camurus, Consultant/speaker, Ipsen, Consultant/speaker, Lilly, Consultant/speaker, Novartis, Consultant/speaker, Pfizer, Consultant/speaker, Recordati, Consultant/speaker. E. Mezősi: Crinetics Pharmaceuticals, Inc., Principal investigator of a research grant, Ipsen, Consultant/speaker, Novartis, Consultant/speaker, Novo Nordisk, Consultant/speaker, Pfizer, Consultant/speaker, Recordati, Consultant/speaker. M. Doknic: Crinetics Pharmaceuticals, Inc., Principal investigator of a research grant, Pfizer, Advisory board member. B. Hu: Crinetics Pharmaceuticals, Inc., Employee and shareholder. D.R. Clemmons: Crinetics Pharmaceuticals, Inc., Consultant. P.J. Trainer: Crinetics Pharmaceuticals, Inc., Employee and shareholder. R.S. Struthers: Crinetics Pharmaceuticals, Inc., Employee and shareholder. A. Krasner: Crinetics Pharmaceuticals, Inc., Employee and shareholder. M. Gadelha: Principal investigator of research grants from Crinetics and Recordati; consultant for Camurus, Crinetics, Ipsen, Novo Nordisk, and Recordati; speaker for Camurus, Ipsen, Novo Nordisk, and Recordati. Paltusotine is a selective, non-peptide, SST2 receptor agonist in development as a once-daily oral treatment for patients with acromegaly or carcinoid syndrome. PATHFNDR-1 was a randomized, double-blind, placebo-controlled trial that evaluated the efficacy and safety of switching to paltusotine in patients whose acromegaly was controlled (IGF-I ≤1.0× ULN) with injected depot somatostatin receptor ligands (SRLs; octreotide LAR or lanreotide depot). PATHFNDR-1 includes a 36-week randomized controlled (RC) phase and a single-arm, open-label extension (OLE; currently ongoing). At the end of the RC phase, IGF-I ≤1.0× ULN was maintained in 83.3% of paltusotine-treated patients versus 3.6% of patients in the placebo control group (P<0.0001). The OLE is evaluating the efficacy and safety of longer-term treatment with paltusotine. IGF-I and GH levels were measured at a central laboratory using validated immunoassays. The Acromegaly Symptom Diary (ASD) is a patient-reported outcome measure that consists of 7 core items (headache, joint pain, sweating, fatigue, leg weakness, swelling, numbness/tingling; score range, 0-70; higher scores indicate greater symptom burden). Fifty-eight patients enrolled in the RC phase (paltusotine, n=30; placebo, n=28); 53 of 58 (91.4%) continued into the OLE (paltusotine in RC, n=27; placebo in RC, n=26). As of the analysis cutoff date (15 Aug 2024), median (range) duration of exposure to paltusotine during the OLE was 72.6 (9.4-120.4) weeks. Fifty patients had efficacy data at Study Week 96 (OLE Week 60; efficacy analysis). Mean (SD) IGF-I was 0.93 (0.22)× ULN at OLE baseline (Week 36) and 0.81 (0.21)× ULN at Week 96 (mean change: -0.11; n=50). At Week 96, mean (SD) IGF-I was similar for patients who received paltusotine (0.82 [0.23]× ULN) or placebo (0.80 [0.20]× ULN) in the RC phase. Mean (SD) GH was 1.0 (1.0) ng/mL at OLE baseline and 1.1 (1.2) ng/mL at Week 96. Mean (SD) ASD score was 10.7 (10.5) at OLE baseline and 12.0 (10.3) at OLE Week 96 (mean change, 1.3; n=42). As of this analysis, the most common AEs during the OLE (reported in >3 patients, as of this analysis) were diarrhea (15.1%), nausea (9.4%), fatigue (7.5%), and urinary tract infection (7.5%). Serious AEs were reported in 6 patients, 1 of which was considered treatment-related (bile duct stone). There was 1 study discontinuation due to an AE: a fatal occurrence of acute combined drug intoxication, which was unrelated to study medication. In conclusion, once-daily oral paltusotine maintained biochemical and symptom control and was well tolerated during long-term treatment in patients with acromegaly who switched from injected SRLs. Support: Crinetics Pharmaceuticals Presentation: Sunday, July 13, 2025
