Results 51 to 60 of about 22,118 (189)

Gemcitabine, Docetaxel, Melphalan, Carboplatin as Part of Sequential Cycles of High‐Dose Chemotherapy With Autologous Hematopoietic Stem‐Cell Rescue for Multiply Relapsed/Refractory Pediatric Germ Cell Tumors

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Although most malignant germ cell tumors (GCTs) are highly curable with cisplatin‐based therapy, options for patients with multiply relapsed/refractory disease remain limited. For this population, we report the first pediatric use of gemcitabine, docetaxel, melphalan, and carboplatin (GemDMC) as part of sequential cycles of high‐dose ...
Maria Frost, Olayinka Okeleji, Amr Elgehiny, Douglas Harrison, Cynthia Herzog, Jeremy Connors, Demetrios Petropoulos, Priti Tewari, Dristhi Ragoonanan, Yago Nieto, Irtiza N. Sheikh   +10 more
wiley   +1 more source

FDA’s implementation of knowledge-aided assessment and structured application for manufacturing assessment of non-sterile solid oral dosage form drug products

AAPS Open
The Knowledge-Aided Assessment and Structured Application (KASA) is an initiative led by the FDA’s Center for Drug Evaluation and Research (CDER) which is launched within CDER’s Office of Pharmaceutical Quality (OPQ). It is a platform designed to conduct
Zhouxi Wang, Jingbo Xiao, Daniel Obrzut, Stelios Tsinontides, Rakhi Shah   +4 more
doaj   +1 more source

CPX‐351 in High‐Risk Relapsed Pediatric Acute Leukemia: Real‐World Phase 1 Data Establishing the FDA‐Approved Dose

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background/Objectives Outcomes for pediatric relapsed/refractory (R/R) acute myeloid leukemia (AML) remain dismal. CPX‐351, a liposomal formulation of cytarabine and daunorubicin, may have less off‐target toxicities than traditional chemotherapies and has shown improved outcomes for adults with newly diagnosed therapy‐related AML.
Jonathan D. Bender, Lauren Pommert, Lori R. Backus, Andrea Bidwell, Courtney Blank, Erin H. Breese, Karen C. Burns, Adam Lane, Lynn H. Lee, Benjamin Mizukawa, Robin E. Norris, Maureen M. O'Brien, Daniel Peck, Christine Phillips, Thomas D. Ryan, Jennifer Young, Michael Absalon, John P. Perentesis   +17 more
wiley   +1 more source

A network of regulatory innovations to improve FDA quality assessments of human drug applications

International Journal of Pharmaceutics: X
A network of regulatory innovations brings a holistic approach to improving the submission, assessment, and lifecycle management of pharmaceutical quality information in the U.S. This dedicated effort in the FDA’s Center for Drug Evaluation and Research (
Russie Tran, Grace Fraser, Adam C. Fisher, Sau L. Lee, Ashley Boam, Stelios Tsinontides, Jennifer Maguire, Lawrence X. Yu, Susan Rosencrance, Steven Kozlowski, Don Henry   +10 more
doaj   +1 more source

Incidence and Risk Factors of Serious Osteonecrosis in Pediatric Acute Lymphoblastic Leukemia: A CYP‐C Population‐Based Study

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Osteonecrosis (ON) is a debilitating complication of acute lymphoblastic leukemia (ALL) therapy. While numerous studies have explored its incidence and associated risk factors, investigations using large‐scale cohorts remain important to characterize ON across heterogeneous populations.
Noémie de Villiers, Chantal Éthier, Caroline Laverdière, Nathalie Alos, Thai Hoa Tran, Marie‐Claude Pelland‐Marcotte   +5 more
wiley   +1 more source

Novel Genetic Risk Factor Identified for L‐Asparaginase‐Induced Pancreatitis in Pediatric Patients With Cancer

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background L‐asparaginase is a critical component in treatment protocols for pediatric acute lymphoblastic leukemia. Acute pancreatitis reactions can necessitate delays and, in some cases, discontinuation of L‐asparaginase, which compromises outcomes.
Edward J. Raack, Wan‐Chun Chang, Miguel Cordova‐Delgado, Spencer J. Anderson, Jessica N. Trueman, Erika N. Scott, Catrina M. Loucks, Shahrad R. Rassekh, Gabriella S. S. Groeneweg, Michelle Higginson, Kathy Li, Fudan Miao, Derek Yau, Donna Johnston, Mounira Ibrahim, Maja Krajinovic, Thai Hoa Tran, Jessica Tewfik, Jean‐François Bussières, Thaïna‐Rafi Jean‐Baptiste, Denis Lebel, Lucie Pecheux, Bina Gyawali, Gregory Guilcher, Gesche Riabowol, Geert ’t Jong, Michelle Staub, Geoff Cuvelier, Kathleen Felton, Sara Khalaj, Michael Rieder, Awatif Abuzgaia, Tamorah Lewis, Himal Ghimire, Paul Nathan, Kerry Goralski, Ketan Kulkarni, Zara Forbrigger, Colin J. D. Ross, Bruce C. Carleton   +39 more
wiley   +1 more source

Outcomes and Surgical Management of Malignant Rhabdoid Tumor of the Kidney: A Report From the Pediatric Surgical Oncology Research Collaborative

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Purpose Malignant rhabdoid tumor of the kidney (MRTK) is a rare, aggressive tumor seen in young children. The optimal timing of resection for locally advanced tumors is not well‐defined. The purpose of this study is to evaluate modern oncologic outcomes and the impact of surgical timing. Methods A multicenter retrospective review was performed
Hannah N. Rinehardt, Elisabeth T. Tracy, Anghela Paredes, Catherine Beckhorn, Harold J. Leraas, Joseph Fusco, Katlyn G. McKay, Nelly‐Ange T. Kontchou, Zachary J. Kastenberg, David W. Hoyt, Jonathan Roach, Emily K. Myers, Nicholas G. Cost, Bhargava Mullapudi, Charles R. Marchese, Amanda R. Jensen, Timothy B. Lautz, Michela Carter, Roshni Dasgupta, John Lundstedt, Joseph G. Brungardt, Lindsay Talbot, Andrew M. Davidoff, Andrew J. Murphy, Jennifer H. Aldrink, Sara Mansfield, Nelson Piche, Annie Le‐Nguyen, Dave R. Lal, Brian T. Craig, Jennifer M. Schuh, Barrett P. Cromeens, Sindhu Mannava, Shannon Castle, Adriana Lopez, Kelsey Mello, Joshua Short, Robin T. Petroze, Shay Rajaval, Grace R. Thompson, Peter Mattei, David H. Rothstein, Elizabeth Fialkowski, Kathryn Fowler, Nathan Martchenke, Barrie S. Rich, Richard D. Glick, Erin G. Brown, Kathleen Doyle, Paige Abril, Natashia Seemann, Jacob Davidson, Claire A. Wilson, Hau D. Le, Devashish Joshi, Michael Stellon, Tamer Ahmed, Alexandra Dimmer, Peter F. Ehrlich, Maya Hammoud, Keyonna Williams, Christa N. Grant, Merit Gorgy, Stephanie F. Polites, Julia Debertin, Danielle B. Cameron, Alyssa Stetson, Eugene S. Kim, William G. Lee, Aaron Barkhordar, Mary Austin, Brian A. Coakley, Anastasia Kahan, Joseph T. Murphy, Michael Pitonak, Chloé Boehmer, Marcus M. Malek   +76 more
wiley   +1 more source

Paraneoplastic Lupus Nephritis in a Child With Neuroblastoma Recurrence

Pediatric Blood &Cancer, EarlyView.
Gabriele Mortari, Caterina Costagliola, Marta Nebiolo, Francesca Parisi, Martina Morini, Katia Mazzocco, Gabriele Gaggero, Stefano Volpi, Stefania Sorrentino, Virginia Livellara   +9 more
wiley   +1 more source

Upacicalcet: A Novel Intravenous Calcimimetic Agent for Patients Undergoing Hemodialysis With Secondary Hyperparathyroidism

Therapeutic Apheresis and Dialysis, EarlyView.
ABSTRACT Secondary hyperparathyroidism (SHPT) is a common complication in patients receiving maintenance dialysis, driven by calcium and phosphate metabolism disturbances. Calcimimetics are central to the management of SHPT by enhancing calcium‐sensing receptor sensitivity and reducing parathyroid hormone secretion.
Fumihiko Koiwa, Daisuke Honda, Seigo Akari, Tadao Akizawa   +3 more
wiley   +1 more source

Adjunctive Therapeutic Plasma Exchange in Refractory Adult‐Onset Still's Disease Complicated by Secondary Macrophage Activation Syndrome: A Single‐Center Experience

Therapeutic Apheresis and Dialysis, EarlyView.
ABSTRACT Introduction Adult‐onset Still's disease (AOSD) complicated by macrophage activation syndrome (MAS) carries substantial mortality. The role of therapeutic plasma exchange (TPE) remains uncertain. Methods We retrospectively analyzed patients with AOSD‐MAS treated with TPE at a single‐center.
Masataka Ueda, Tomohiro Saito, Masahide Mizobuchi, Tomoki Shimomura, Saki Kawamori, Mitsuru Kawanishi, Natsuko Hattori, Jo Sugiyama, Satoshi Tsubata, Hanami Horiguchi, Kazuki Abe, Nao Iida, Yuki Kajio, Nao Oguro, Ryo Yanai, Hirokazu Honda   +15 more
wiley   +1 more source