Results 51 to 60 of about 11,812,248 (285)

Initial Psychometric Evaluation of the Barth Syndrome Symptom Assessment (BTHS-SA) for Adolescents and Adults in a Phase 2 Clinical Study

Orphanet Journal of Rare Diseases
Background Barth syndrome (BTHS) is a rare, X-linked disorder that stems from mutations in the TAFAZZIN (TAZ) gene with varying disease severity among patients.
Chad Gwaltney, Alan Shields, Emily Love, Sarah Ollis, Jonathan Stokes, Iyar Mazar, Ethan Arenson, Anthony Aiudi, R. J. Wirth, Carrie Houts   +9 more
doaj   +1 more source

Pediatric Idiopathic Multicentric Castleman Disease Is Often Severe But Responsive to Siltuximab

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Idiopathic multicentric Castleman disease (iMCD) is a potentially fatal immunologic disorder marked by widespread lymphadenopathy and inflammation. Siltuximab, an interleukin‐6 (IL‐6) inhibitor, is the only FDA‐approved treatment for adult patients with iMCD.
Bridget Austin, Sheila K. Pierson, Kelley C. Dacus, Mateo Sarmiento Bustamante, Saishravan Shyamsundar, Larissa Borys, Kayla McHugh, Abiola Irvine, Yoojin Jo, Adam Bagg, Mary Jo Lechowicz, Daisy Alapat, Amy Chadburn, Megan S. Lim, Gordan Srkalovic, Frits van Rhee, David C. Fajgenbaum, Joshua D. Brandstadter   +17 more
wiley   +1 more source

Effectiveness of a Values Education Program for Preschool Students: A Teacher-Based Evaluation Study [PDF]

The purpose of this study is to investigate the effectiveness of the Preschool Values Education Program, which was developed in line with the values identified through a needs analysis and prepared in accordance with the Taba Curriculum Development Model.
Kayıran, Derya, Bağçeci, Birsen
core   +1 more source

Justifying score interpretation thresholds for the Asthma Control Questionnaire (ACQ-5) in a contemporary asthma patient population

Journal of Patient-Reported Outcomes
Background The 5-item Asthma Control Questionnaire (ACQ-5) is widely used in clinical trials to assess the adequacy of asthma symptom control and any changes in asthma control as a result of treatment.
George Skingley, Mattea Orsini, Jessica Middlehurst, Jessica Flynn, James Brookes, Hermione Blackwood, Nathan Clarke, Rebecca Williams-Hall, Benoit Arnould, Renata Martincova, Jane Wells   +10 more
doaj   +1 more source

Referral Patterns and Diagnostic Timeliness in Pediatric Cancer: A Hospital‐Based Study in Indonesia

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Timely diagnosis and treatment are critical for improving survival among children with cancer. In low‐ and middle‐income countries (LMICs), delays are common and may be influenced by fragmented referral pathways and diagnostic limitations.
Nur Melani Sari, Regina Hadi Putri, Fatimah Assyifa, Judith Spijkerman, Gertjan Johannes Kaspers, Susi Susanah   +5 more
wiley   +1 more source

Language, Culture, and Cancer: Qualitative Insights Into Communication Disparities Among Spanish‐Speaking Caregivers of Children With Cancer

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Latino children are projected to make up nearly one‐third of United States (US) children by 2060, and many of their caregivers speak Spanish. Prior survey research has documented communication difficulties for Spanish‐speaking caregivers of children with cancer, but contemporary qualitative data are limited.
Jenny Ruiz, Alejandra Curbelo‐Paz, Sonia Morales, Elizabeth Contreras, Amy Houtrow, Scott H. Maurer, Judy C. Chang   +6 more
wiley   +1 more source

Patient Experience of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP): A Qualitative Exploration of Signs, Symptoms, and Health-Related Quality of Life Impacts

Neurology and Therapy
Introduction Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare type of autoimmune neuropathy, characterized by signs of distal and proximal weakness of the upper and lower limbs, sensory dysfunction, absent or diminished tendon reflexes,
Anna Roberts, Natasha Griffiths, Kieran Thiara, Sophie Wallace, Alyson L. Young, Nicola Williamson, Adam Gater, Omar Saeed, Charles Minor, Natalia Hawken   +9 more
doaj   +1 more source

Use of the European Organisation for Research and Treatment of Cancer multiple myeloma module (EORTC QLQ-MY20): a review of the literature 25 years after development

Blood Cancer Journal, 2023
The European Organisation for Research and Treatment of Cancer Quality of Life Multiple Myeloma Questionnaire (EORTC QLQ-MY20) was developed in 1996 to assess health-related quality of life (HRQoL) in patients with multiple myeloma. Since its development
K. Forde, K. Cocks, J. R. Wells, I. McMillan, C. Kyriakou, EORTC Quality of Life Group   +5 more
doaj   +1 more source

Biphasic Clinical Course of Pediatric Cytomegalovirus‐Associated Immune Thrombocytopenia: Early Poor Treatment Response but Favorable Long‐Term Outcomes

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Cytomegalovirus (CMV) is a recognized trigger of immune thrombocytopenia (ITP); however, its incidence and impact on disease course in children remain controversial. Therefore, we aimed to characterize the clinical course of pediatric patients with CMV‐associated ITP.
Oded Gilad, Ohad Atia, Osama Tanous, Dafna Brik Simon, Michal Dvori, Miriam Cohen, Tracie A. Goldberg, Efraim Bilavsky, Joanne Yacobovich, Orna Steinberg‐Shemer   +9 more
wiley   +1 more source

Qualitative exploration of women’s experiences of vasomotor symptoms to support the content validity of patient-reported outcomes

Journal of Patient-Reported Outcomes
Background Frequency and severity of vasomotor symptoms (VMS; hot flashes) associated with menopause significantly impact women’s health-related quality of life (HRQoL). Treatment benefit in VMS clinical trials is assessed using patient-reported outcome (
Claudia Haberland, Melissa Barclay, Sophie Whyman, Asha Lehane, Adam Gater, Christoph Gerlinger, Christian Seitz, Maja Francuski, Nils Schoof, Andrew Trigg, Helena Bradley   +10 more
doaj   +1 more source