While the event‐free survival (EFS) of children treated for acute lymphoblastic leukaemia (ALL) has improved greatly in the last decades, the EFS for patients diagnosed with ALL before the age of one is still under 50%. This outcome further decreases when infants have a rearrangement in the gene encoding histone‐lysine N‐methyltransferase 2A (KMT2A ...
Tirsa de Kluis +5 more
wiley +1 more source
Pentoxifylline dose finding trial in preterm neonates with suspected late onset sepsis (PTX‐trial)
Aim The aim of this study (PTX‐trial) is to determine the optimal dose of pentoxifylline (PTX) in preterm neonates (gestational age < 30 weeks) with (suspected) late onset sepsis (LONS). Methods The PTX‐trial is a prospective multicentre open‐label sequential dose‐optimization study with an adapted continual reassessment method.
Serife Kurul +7 more
wiley +1 more source
Patient‐reported outcomes (PROs) are essential for assessing clinical benefit and tolerability from the patient's perspective. In oncology trials, PRO data analysis often involves responder and time‐to‐event analyses, which depend on predefined thresholds to determine clinically meaningful change.
Anna Margarete Maria Thurner +5 more
wiley +1 more source
Safety and Tolerance of a Novel MMR (Measles, Mumps, and Rubella) Vaccine in Indian Children: A Real-World Evidence Study. [PDF]
Pula JR +10 more
europepmc +1 more source
Introduction In recent years, the treatment of spinal muscular atrophy (SMA), a rare disease, has significantly progressed, improving patients' survival and overall quality of life. However, current SMA treatments are expensive, and some (nusinersen) are very inconvenient for patients.
Andrej Belančić +4 more
wiley +1 more source
Retrospective analysis of the tolerability and effectiveness of rimegepant for the acute treatment of migraine in adolescents. [PDF]
Tourlas K +4 more
europepmc +1 more source
PROMISE‑MINO: A Prospective, Open‑Label, Multicenter, Single‑Arm, Phase IV Study of the Safety, Local Tolerability, and Efficacy of Topical Minocycline Hydrochloride 4% Gel in Indian Patients With Moderate‑to‑Severe Acne Vulgaris. [PDF]
Viswanath V +13 more
europepmc +1 more source
Single-ascending and multiple-ascending dose study of the pharmacokinetics, safety, and tolerability of BV100 (rifabutin for infusion) in healthy volunteers. [PDF]
Kemmer C +6 more
europepmc +1 more source
Revisiting the Ethics of Urate‐Lowering Therapy Clinical Trials for Gout Management
Arthritis &Rheumatology, EarlyView.
Lisa K. Stamp, Dien Ho, Nicola Dalbeth
wiley +1 more source

