Results 81 to 90 of about 559,639 (268)

Real-life impacts of olipudase alfa: experiences of adults receiving enzyme replacement therapy for acid sphingomyelinase deficiency—results from an international survey study

Orphanet Journal of Rare Diseases
Background Acid sphingomyelinase deficiency (ASMD) is a rare lysosomal storage disorder caused by SMPD1 mutations, resulting in sphingomyelin accumulation and diverse manifestations.
Adel Sabet Morsy, Solomon Mbua, Toni Mathieson, Justin Hopkin, Shaun Bolton   +4 more
doaj   +1 more source

Impact of Metastatic Patterns on Survival and Response to Therapy in Neuroblastoma

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background While the presence of metastases in neuroblastoma (NB) is a well‐established prognostic factor, the clinical significance of dissemination patterns and tumour burden and their impact on response and survival remains poorly understood.
Mariona Morell‐Daniel, Santiago Pérez‐Hoyos, Aroa Soriano, Ana Coma, Amalia Varo, Raquel Hladun, Gabriela Guillén, Soraya Mico, Marta Garrido, Lourdes Rubio, Alba Rubio‐San‐Simón, Ana Minguez, Ricardo López‐Almaraz, Laura Illade, Miguel F. Segura, Lucas Moreno   +15 more
wiley   +1 more source

The Future of Pediatric Hematology‐Oncology Fellowship Training: Reflections on the American Board of Pediatrics Training Model

Pediatric Blood &Cancer, EarlyView.
Scott C. Borinstein, Scott Moerdler, Alexander A. Boucher, Stacy L. Cooper, Rachel A Egler, Erika Friehling, Richard Ho, Catherine McGuinn, Kathryn S. Sutton, Stacey G. Zahler, Leslie Kersun, Jennifer Kesselheim   +11 more
wiley   +1 more source

Sociodemographic Factors Associated With Later Stage at Diagnosis of Pediatric Germ Cell Tumors: A Report From Children's Oncology Group Registries ACCRN07 and APEC14B1

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Germ cell tumors (GCTs) often arise in the ovaries and testes (extracranial) but can also develop in the brain (intracranial). We examined the relationship of individual, family, and community‐level socioeconomic status (SES) with stage of disease at diagnosis in a cohort of pediatric patients with GCT from Children's Oncology Group
Heydon K. Kaddas, Judy Y. Ou, Todd Alonzo, Logan G. Spector, Negar Fallahazad, Emily Owens, Anne C. Kirchhoff, Adam L. Green   +7 more
wiley   +1 more source

Comparison of the American PPCM Registry Data With International Registries

Journal of the American College of Cardiology, 2016
We congratulate McNamara et al. [(1)][1] for these exciting new data on a cohort of 100 women diagnosed with peripartum cardiomyopathy (PPCM). This study adds important information with regard to ethnic differences and prognosis as the current management allows it.
Hilfiker-Kleiner, Denise, Sliwa-Hahnle, Karen, Bauersachs, J.   +2 more
openaire   +4 more sources

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

Epidemiology and treatment of malignant ovarian germ cell and sex cord stromal tumors in germany: a population-based cancer registry study from 2016 – 2021

Scientific Reports
Malignant ovarian germ cell tumors (MOGCT) and ovarian sex cord stromal tumors (SCST) are rare ovarian tumors. Since 2013, all German federal states are obliged to record data on diagnosis, pathology, therapy, and clinical course of tumor diseases.
Dennis Jung, Sinaida Branscheidt, Ian Wittenberg, Andrea Schmidt-Pokrzywniak, Daniela Piontek, Tonia Brand, Mike Klora, Sylke Ruth Zeissig, Nikola Beck, Anita Feller, Silke Hermann, Josephine Kanbach, Sabine Luttmann, Ron Pritzkuleit, Bettina Braun, Cynthia Erb, Henrik Kusche, Gabriele Robers, Kerstin Weitmann, Khalid Abnaof, Soo-Zin Kim-Wanner, Daniela Reil, Anne von Rüsten, Florian Oesterling, Andreas Stang, Cécile Ronckers, Claudia Bremensdorfer, Christina Justenhoven, Annette Hasenburg   +28 more
doaj   +1 more source

Psoriatic Arthritis Registries

The Journal of Rheumatology Supplement, 2015
The introduction of new biological drugs for the treatment of rheumatoid arthritis and spondyloarthritis has led to the creation of a number of registries in Europe and the United States. Most of them are sponsored by national rheumatology societies, and provide information that is useful in clinical practice concerning the clinical characteristics ...
Sarzi-Puttini, Piercarlo, Varisco, Valentina, Ditto, Maria Chiara, Benucci, Maurizio, Atzeni, Fabiola   +4 more
openaire   +3 more sources

Mapping Childhood Cancer Care Services in Oceania: Methodological Lessons From the SIOP Global Mapping Programme

Pediatric Blood &Cancer, EarlyView.
ABSTRACT The International Paediatric Oncology Society Global Mapping Programme aims to collect data on global paediatric oncology services by continent, with Africa and Latin America completed. This study reports on the methodology and lessons learned for Oceania, a unique continent with resource disparity across countries, multiple small island ...
Trisha Soosay Raj, Christy Noble, Amy Gray, Daniel C. Moreira, Jennifer Lowe, Maite Gorostegui, Gwenda Anga, Andrew Soma, Milena Lay Dos Santos, Siobhan Cross, Erin Luttrell, Michael Sullivan, Julie Cayrol, Julia Challinor, Neil Ranasinghe, Jordana McLoone, Claire E. Wakefield   +16 more
wiley   +1 more source