Results 51 to 60 of about 202,059 (264)

Childhood Acute Lymphoblastic Leukemia Survival in Western Kenya: Reduction in Early Deaths and Treatment Abandonment

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background An earlier study on children diagnosed with acute lymphoblastic leukemia (ALL) at Moi Teaching and Referral Hospital (MTRH) in Kenya reported a low event‐free survival (EFS), excess treatment abandonment, and high induction mortality.
Gilbert Olbara, George Bogonko, Festus Njuguna, Sandra Langat, Martha Kipngetich, Dennis Njenga, Gertjan Johannes Kaspers, Terry Allen Vik   +7 more
wiley   +1 more source

Pathogenicity of Hypertrophic Cardiomyopathy Variants [PDF]

Circulation: Cardiovascular Genetics, 2017
The challenges of correctly interpreting rare variants identified via genetic testing of patients with hypertrophic cardiomyopathy (HCM) are universal and ever present. HCM genetic testing has been offered for almost 2 decades, yet in spite of the leaps forward in our understanding, the genetic underpinnings of this disease remain elusive in many ...
Jodie, Ingles, Charlotte, Burns, Birgit, Funke   +2 more
openaire   +2 more sources

Immunoglobulin Depletion and Recovery Following Blinatumomab in Infants With KMT2A‐Rearranged ALL

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Adding blinatumomab to standard chemotherapy for infants with KMT2A‐rearranged acute B‐cell lymphoblastic leukemia (KMT2A‐r B‐ALL) improves outcomes. Although blinatumomab impairs immunoglobulin G (lgG) production, increasing infection susceptibility, IgG recovery remains poorly understood.
Miguel Vieira Martins, Paola de Lorenzo, Rishi S. Kotecha, Andishe Attarbaschi, Gabriele Escherich, Karsten Nysom, Jan Stary, Alina Ferster, Benoit Brethon, Franco Locatelli, Martin Schrappe, Peggy E. Scholte‐van Houtem, Maria G. Valsecchi, Rob Pieters, Inge M. van der Sluis   +14 more
wiley   +1 more source

Ovarian Sex Cord Stromal Tumors in Children and Adolescents—The European Standard Clinical Practice Recommendations

Pediatric Blood &Cancer, EarlyView.
ABSTRACT As part of the European Cooperative Study Group for Paediatric Rare Tumours initiative, we developed standard clinical practice guidelines for ovarian sex cord stromal tumors, based on comprehensive national and international cohort analyses, literature review, and a final expert consensus conference.
Dominik T. Schneider, Kris‐Ann Schultz, Nora Schnitzler, Michael Abele, Tal Ben‐Ami, Ewa Bien, Gianni Bisogno, Ines B. Brecht, Maja Cesen, Andrea Ferrari, Michaela Kuhlen, Malgorzata A. Krawczyk, Ricardo Lopez‐Almaraz, Yves Reguerre, Calogero Virgone, Daniel Orbach   +15 more
wiley   +1 more source

Natural Killer Cells in Paediatric Soft Tissue Sarcomas: A Systematic Review

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Paediatric soft tissue sarcomas (pSTS) are a rare and heterogeneous group of malignant tumours arising in tissues of mesenchymal origin. The role of natural killer (NK) cells in pSTS remains poorly understood, with evidence fragmented across small preclinical studies and early‐phase clinical trials.
Raya Dean, Lara V. Graham, Christina Putnam, Rosa Gomes Alves Martins, Daniel T. Harding, Tim J. Underwood, Zoë S. Walters, Matthew D. Blunt   +7 more
wiley   +1 more source

Animal‐Assisted Activities With Therapy Dogs in Pediatric Oncology: A Multicenter Survey of the Current Status, Implementation, and Challenges in Germany, Austria, and Switzerland

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Animal‐assisted activities (AAAs) with therapy dogs have shown positive effects on patient well‐being and quality of life in various areas of medicine, including pediatric oncology. However, research on this topic is limited. The aim of this study is to present the current status of AAA in pediatric oncology in Germany, Austria, and
Jan‐Marius Wedig, Birgit Burkhardt, Christof M. Kramm, Kira Ostermann, Sarah C. Goretzki, Uta Dirksen, Nico Kurpiers, Michael M. Schündeln   +7 more
wiley   +1 more source

Spectrum of genetic mutations among Iranian patients with primary hyperoxaluria type 1

Journal of Clinical and Translational Endocrinology Case Reports
Primary hyperoxaluria type 1 (PH1) is a genetic disorder by defect in the liver peroxisomal enzyme alanine-glyoxylate aminotransferase (AGT). Here, we report the clinical and molecular data of four Iranian patients with PH1.
Mohadeseh Fathi, Sheyda Khalilian, Nakysa Hooman, Mostafa Sharifian, Saeed Talebi, Faezeh Sherafat, Fatemeh Fazeli, Mohammad Miryounesi, Soudeh Ghafouri-Fard   +8 more
doaj   +1 more source

Clinical and molecular findings in three Japanese patients with N-acetylneuraminic acid synthetase-congenital disorder of glycosylation (NANS-CDG)

Scientific Reports, 2022
We report clinical and molecular findings in three Japanese patients with N-acetylneuraminic acid synthetase-congenital disorder of glycosylation (NANS-CDG).
Yohei Masunaga, Gen Nishimura, Koji Takahashi, Tomiyuki Hishiyama, Masatoshi Imamura, Kenichi Kashimada, Machiko Kadoya, Yoshinao Wada, Nobuhiko Okamoto, Daiju Oba, Hirofumi Ohashi, Mitsuru Ikeno, Yuko Sakamoto, Maki Fukami, Hirotomo Saitsu, Tsutomu Ogata   +15 more
doaj   +1 more source

On Hierarchical Composite Endpoints in Pediatric Cancer Supportive Care: Illustrative Examples From Two Multi‐Center Phase‐III Randomized Clinical Trials

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Pediatric supportive care clinical trials often involve multiple clinically important outcomes, complicating trial interpretation. Hierarchical composite endpoints (HCEs) provide a framework to integrate key outcomes according to clinical importance.
Willem H. Collier, Mark Zobeck, Adam J. Esbenshade, Christopher C. Dvorak, Lillian Sung, David Freyer, Sarah Alexander, Etan Orgel, Nicole J. Ullrich, Zach Prudowsky, Brian Fisher, Caitlin W. Elgarten   +11 more
wiley   +1 more source

Modeling Pathogenic Variants in the RNA Exosome.

RNA & disease (Houston, Tex.), 2020
<p>Published - <a href="/records/k2m6f-9w889/files/1166-Manuscript-1589-1-10-20200617.pdf?download=1">1166-Manuscript-1589-1-10-20200617.pdf</a></p>
de Amorim, Julia, Slavotinek, Anne, Fasken, Milo B., Corbett, Anita H., Morton, Derrick J.   +4 more
openaire   +3 more sources