Results 91 to 100 of about 13,796 (248)

Pharmacokinetics, Effectiveness and Safety of Certolizumab Pegol in Children and Adolescents With Active Juvenile Idiopathic Arthritis: 9+‐Year Results From a Multicenter, Open‐Label Study

Arthritis &Rheumatology, EarlyView.
Objective The purpose of this study was to assess the pharmacokinetics, effectiveness, safety, and immunogenicity of certolizumab pegol (CZP) in polyarticular‐course juvenile idiopathic arthritis (pcJIA). Methods Pediatric Arthritis Study of Certolizumab Pegol (NCT01550003), a multicenter, open‐label study, enrolled patients aged 2 to 17 years with ...
Hermine I. Brunner, Lyudmila Grebenkina, Irina Nikishina, Ekaterina Alexeeva, Vyacheslav Chasnyk, Carlos Abud‐Mendoza, Nadina Rubio‐Pérez, Heinrike Schmeling, María del Rocio Maldonado‐Velázquez, Diane Brown, Andrew Zeft, Esi Morgan, C. Egla Rabinovich, Daniel Kingsbury, Hulya Bukulmez, Beth Gottlieb, Thomas Griffin, Lisa Imundo, Stacey Tarvin, Earl Silverman, Laura Shaughnessy, Saori Shimizu, Paulette Williams, Rocío Lledó‐García, Gauri Utturkar, Baran Ufuktepe, Alberto Martini, Daniel J. Lovell, Nicolino Ruperto, the Paediatric Rheumatology International Trials Organisation (PRINTO); the Pediatric Rheumatology Collaborative Study Group (PRCSG), Hermine I. Brunner, Lyudmila Grebenkina, Irina Nikishina, Ekaterina Alexeeva, Vyacheslav Chasnyk, Carlos Abud‐Mendoza, Nadina Rubio‐Pérez, Heinrike Schmeling, María del Rocio Maldonado‐Velázquez, Diane Brown, Andrew Zeft, Esi Morgan, C. Egla Rabinovich, Daniel Kingsbury, Hulya Bukulmez, Beth Gottlieb, Thomas Griffin, Lisa Imundo, Stacey Tarvin, Earl Silverman, Alberto Martini, Daniel L. Lovell, Nicolino Ruperto   +52 more
wiley   +1 more source

Switching disease‐modifying therapies in patients with spinal muscular atrophy: A systematic review on effectiveness outcomes

British Journal of Clinical Pharmacology, EarlyView.
With multiple disease‐modifying therapies now available, treatment switching has become an important clinical consideration in the management of spinal muscular atrophy (SMA). While some switches are prompted by suboptimal clinical response, more commonly they are driven by treatment burden, convenience, or adverse events.
Andrej Belančić, Elvira Meni Maria Gkrinia, Patrick Eustaquio, Andrea Katrin Faour, Dinko Vitezić   +4 more
wiley   +1 more source

Effect of developmental changes on pharmacokinetics of drugs used in the treatment of infant acute lymphoblastic leukaemia—A comprehensive review

British Journal of Clinical Pharmacology, EarlyView.
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, Leiah J. Brigitha, Lidwien M. Hanff, Alwin D. R. Huitema, Inge M. van der Sluis, Meta H. M. Diekstra   +5 more
wiley   +1 more source

Oral Azacitidine Maintenance After CPX‐351 in Transplant‐Ineligible Patients With Therapy‐Related or Myelodysplasia‐Related Acute Myeloid Leukemia

American Journal of Hematology, EarlyView.
Caroline Fileni, Edmond Chiche, David Swoboda, Alexander Coltoff, Rami Komrokji, Geoffrey Uy, Sarah Bertoli, Lenaig Le Clech, Mael Heiblig, Ludovic Gabellier, Patrick Lawrence, Lydia Kretschmer, Christoph Röllig, A. H. Wei, Fabio Guolo, Roberto Massimo Lemoli, Jeffrey Lancet, David Sallman, Thomas Cluzeau   +18 more
wiley   +1 more source

Efficacy, safety and cost‐effectiveness of CAR‐T therapy

British Journal of Clinical Pharmacology, EarlyView.
CAR T‐cells demonstrate high efficacy in blood cancers, including ALL, MM and DLBCL. Innovations target solid tumours despite challenges such as antigen escape. Combination therapies enhance the delivery and infiltration of CAR T cells. Toxicity, cost and resistance remain major barriers to clinical use.
Emina Karahmet Sher, Amina Džidić‐Krivić, Emma Pinjic, Nejra Selak, Kanita Omerbasic, Alexandar Chupin, Andrej Belancic, Almir Fajkic   +7 more
wiley   +1 more source

Exome sequencing in 116 patients with inherited thrombocytopenia that remained of unknown origin after systematic phenotype-driven diagnostic workup. [PDF]

Haematologica, 2023
Marconi C, Pecci A, Palombo F, Melazzini F, Bottega R, Nardi E, Bozzi V, Faleschini M, Barozzi S, Giangregorio T, Magini P, Balduini CL, Savoia A, Seri M, Noris P, Pippucci T.   +15 more
europepmc   +1 more source

Inherited thrombocytopenias

Haematologica, 2007
Alan T. Nurden, Paquita Nurden
openaire   +3 more sources

From anthelmintic to neuro‐oncology: A systematic review of mebendazole repurposing for brain tumour therapy

British Journal of Clinical Pharmacology, EarlyView.
Abstract Aim Mebendazole (MBZ), a benzimidazole anthelmintic with established clinical use, has emerged as a repurposing candidate for primary brain tumours due to its multimodal anticancer actions and central nervous system penetrance. This systematic review synthesizes preclinical and clinical evidence evaluating MBZ's efficacy, mechanisms of action ...
Ciara B. Blum, Milli McMenamin, Tessa Khoo, John M. Edwin, Prathibha Jose, Liam A. O’Callaghan   +5 more
wiley   +1 more source

Clinical and laboratorial characterization of a cohort of patients with hereditary platelet disorders in Brazil

Hematology, Transfusion and Cell Therapy
Introduction: Inherited platelet disorders are rare conditions characterized by altered platelet function and/or reduced platelet counts. Diagnosing these disorders is challenging and may result in delays, misdiagnosis, and inappropriate treatment.
Letícia Dalla Vecchia Grassi, Erica Okazaki, Cynthia Rothschild, Paula Villaça, Fernanda Andrade Orsi, Bianca Stefanello   +5 more
doaj   +1 more source

Decoding RNA regulation: Challenges and opportunities for RNA‐based therapies in Europe

British Journal of Clinical Pharmacology, EarlyView.
Abstract RNA‐based medicinal products represent a promising frontier in personalised medicine, offering sequence‐specific disease targeting at various molecular levels, yet their clinical translation in the European Union (EU) may be hindered by regulatory uncertainty around definitions and evidence requirements; this study therefore aims to identify ...
Olivia C. Lewis, Christine Leopold, Joyce M. Hoek, Raymond M. Schiffelers, Marie L. De Bruin   +4 more
wiley   +1 more source