Predictive value of MRD monitoring by BCR clonality in the ZUMA-7 randomized clinical trial for CAR T therapy in r/r lymphoma. [PDF]
British Journal of Haematology, Volume 208, Issue 5, Page 1850-1853, May 2026.
Miles B +5 more
europepmc +2 more sources
Clinical outcomes and safety in patients with lower-risk myelodysplastic syndromes treated with imetelstat: Substudy of the phase 3 IMerge trial. [PDF]
British Journal of Haematology, Volume 208, Issue 2, Page 737-741, February 2026.
Komrokji RS +20 more
europepmc +2 more sources
Terlipressin for Hepatorenal Syndrome in Patients With Early-Stage Acute-on-Chronic Liver Failure. [PDF]
ABSTRACT Background & Aims Hepatorenal syndrome‐acute kidney injury (HRS‐AKI) is a life‐threatening complication of decompensated cirrhosis. The US Food and Drug Administration approved terlipressin use for HRS‐AKI based on the CONFIRM study, which demonstrated a significant improvement in HRS reversal with terlipressin versus placebo.
Rockey DC +7 more
europepmc +2 more sources
Maralixibat Reduces Serum Bile Acids and Improves Cholestatic Pruritus in Adolescents With Alagille Syndrome. [PDF]
ABSTRACT Background Alagille syndrome (ALGS) is a multisystem cholestatic disorder. Maralixibat is approved for the treatment of cholestatic pruritus in ALGS with limited data in adults. Methods Participants were included if they received ≥ 2 doses of maralixibat at age ≥ 16 years in one of the three previously published maralixibat ALGS clinical ...
Hirschfield G +5 more
europepmc +2 more sources
Phase II clinical trial of nirogacestat in patients with relapsed ovarian granulosa cell tumours. [PDF]
Evidence suggests NOTCH activation is among the survival and proliferation pathways interacting with FOXL2 c.402C > G (p.Cys134Trp) mutation in granulosa cell tumours (GCT). This Phase II clinical trial of nirogacestat in GCT achieved its enrolment target in < 1 year and primary analysis within 2 years.
Grisham RN +20 more
europepmc +2 more sources
MEDINFO‐maailmankongressi Kapkaupungissa
Toimitus
doaj +1 more source
A model of academic-practice collaboration for facilitating informatics capacity and building a learning health system framework in public health. [PDF]
Abstract Background and Objective The data modernization initiative (DMI) is a multi‐year, multi‐billion‐dollar endeavor toward a robust public health information infrastructure. The various DMI projects (interoperability, analytics, workforce, governance) present an opportunity for a learning health system (LHS) framework in public health.
Rajamani S +8 more
europepmc +2 more sources
Bradycardia and Other Arrhythmias in Patients With Hepatorenal Syndrome-Acute Kidney Injury Following Terlipressin Treatment: A Pooled Analysis of Three North American Phase III Clinical Studies. [PDF]
In a pooled analysis, bradycardia was observed in 6% of terlipressin‐treated patients but rarely required a dose interruption or reduction. Arrhythmia serious adverse event incidences were low and similar in both treatment groups. Routine intensive cardiac monitoring for the detection of arrhythmias may not be necessary during terlipressin ...
Bajaj JS +6 more
europepmc +2 more sources
Objective Delandistrogene moxeparvovec is approved in the USA for the treatment of ambulatory patients (4–5 years) with Duchenne muscular dystrophy. ENDEAVOR (SRP‐9001‐103; NCT04626674) is a single‐arm, open‐label study to evaluate delandistrogene moxeparvovec micro‐dystrophin expression, safety, and functional outcomes following administration of ...
Craig M. Zaidman +17 more
wiley +1 more source

