Results 71 to 80 of about 757,641 (252)

Two Faces of NOTCH1 in Childhood Lymphoblastic T‐Cell Neoplasia: Prognostic Divergence of Mutational and Structural Aberrations

Pediatric Blood &Cancer, EarlyView.
ABSTRACT In pediatric patients, T‐cell lymphoblastic lymphoma (T‐LBL) survival exceeds 80%. Relapse remains associated with limited curative options. Frontline treatment is largely extrapolated from T‐cell acute lymphoblastic leukemia (T‐ALL) treatment, reflecting the ongoing debate, whether both entities represent distinct diseases or variants within ...
Marie C. Heider, Amelie Alfert, Marc Hotfilder, Birgit Burkardt, Marcel te Vrugt   +4 more
wiley   +1 more source

Blinatumomab Utilization in Pediatric B‐Cell Acute Lymphoblastic Leukemia: Experience From the Mountain West

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Blinatumomab is a bispecific T‐cell engager approved for the treatment of pediatric B‐cell acute lymphoblastic leukemia (B‐ALL). Outpatient home infusion reduces hospitalization burden and optimizes resource utilization, but is logistically challenging.
Angela Parra del Riego, Kellee Parker, William C. Thomsen, James B. Ford, Mallorie B. Heneghan, David Spencer Mangum, Jennifer L. McNeer, Luke D. Maese, Claire E. F. Miller, Richard Lemons, Taumoha Ghosh   +10 more
wiley   +1 more source

Is It Possible to Have Cheaper Drugs and Preserve the Incentive to Innovate: Reforming the Drug Approval Process According to Market Principles [PDF]

This paper argues that drugs are expensive not because of a lack of competition among research-based pharmaceutical companies, but because of a lack of competition in the drug approval process.
Corinne Sauer, Robert Sauer
core  

Desirable Characteristics of Hepatitis C Treatment Regimens: A Review of What We Have and What We Need. [PDF]

, 2016
There have been dramatic advancements in the treatment of chronic hepatitis C (HCV) infection. This is largely due to the approval of several direct-acting antiviral agents (DAAs) from a variety of medication classes with novel mechanisms of action ...
Bidell, Monique R, Faragon, John, McLaughlin, Milena, Morse, Caroline, Patel, Nimish   +4 more
core   +2 more sources

Clinical and Biological Features of Response in Resistant Neuroblastoma to 131I‐Metaiodobenzylguanidine Radiotherapy in the Anti‐GD2 Immunotherapy Era

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background 131I‐metaiodobenzylguanidine (131I‐MIBG) radiotherapy is a key treatment for relapsed and refractory (R/R) neuroblastoma (NB). Patients with R/R disease treated in the modern era are increasingly exposed to anti‐GD2 immunotherapy, which exerts selective pressure and may modify both tumor cell state and microenvironment.
Benjamin J. Lerman, Katherine K. Matthay, Fabienne Hollinger, Catriona Black, Ann A. Lazar, Miguel Pampaloni, Steven G. DuBois, Kieuhoa T. Vo   +7 more
wiley   +1 more source

Surrogate Endpoints in Pivotal Clinical Trials for Drug Approval in Japan Compared to the United States

Clinical and Translational Science
Regulatory guidance documents exist on surrogate endpoints in the United States. In Japan, there are no established rules or guidance regarding the use of surrogate endpoints, and various aspects remain unclear.
Yuko Yamamoto, Harumi Kosaka, Hanami Nojiri, Hideki Maeda   +3 more
doaj   +1 more source

Global Regulatory Perspectives on Clinical Data Management: A Comparative Review of Various Regulatory Agencies

National Board of Examinations Journal of Medical Sciences
Background: Clinical trials are conducted with a set of ethical standards, patient safety measures, and scientific scrutiny. Clinical Data Management Systems have evolved over time, shaped by historical milestones, technological advancement, and ...
Thrupthi D L, Harshwardhan Patil, Rajath K Bharadwaj, Sameeksha K, Srikant MS   +4 more
doaj   +1 more source

Hospitalization Through Families’ Eyes: Comparing Inpatient Care Quality for Children With Sickle Cell Disease and Cystic Fibrosis in Canada

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Sickle cell disease (SCD) is a chronic, inherited hemoglobinopathy that requires frequent hospitalization for disease‐related complications. Canadian data on inpatient care is limited. This study compared caregiver‐reported hospital experiences of children with SCD to those with cystic fibrosis (CF), a chronic, autosomal recessive ...
Hailey M. Zwicker, Kyle Kemp, Perri Tutelman, Sharon H. J. Hou, Paul Fairie, Maria Santana, Gregory M. T. Guilcher, Taryn Fay‐McClymont, Glenda Bendiak, Wendy Pelletier, Nicola Wright, Fiona Schulte   +11 more
wiley   +1 more source

Real-World Outcomes of Incurable Cancer Patients Treated with Unlisted Anticancer Treatments in an Academic Center in Quebec, Canada

Current Oncology
Medical oncology is a rapidly evolving field, with new medications being discovered yearly, contributing to increased survival rates. However, accessing drugs in a timely manner can be challenging. In Quebec, Canada, a physician can prescribe an unlisted
Adam Miller, Francois Panet, Victoria Korsos, Wilson H. Miller, Gerald Batist   +4 more
doaj   +1 more source

Ethnic Differences in the Association Between SOD2 rs4880 and Hepatotoxicity in Pediatric Acute Lymphoblastic Leukemia: A Report From the REDIAL Consortium

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Treatment‐associated hepatotoxicity (TAH) is a common complication of pediatric acute lymphoblastic leukemia (ALL) treatment, but genetic risk factors remain poorly understood. We evaluated the SOD2 rs4880 variant in 544 children with ALL at Texas Children's Hospital. After adjusting for demographic and clinical covariates, the rs4880 C allele
Emily J. Mason, John P. Woodhouse, Joanna S. Yi, M. Monica Gramatges, Olga A. Taylor, Veronica A. Morales, Shalini Dhamodharan, Marley Roberts, Karen R. Rabin, Philip J. Lupo, Michael E. Scheurer, Van Huynh, Steven D. Mittelman, Etan Orgel, Austin L. Brown   +14 more
wiley   +1 more source