Download full-text PDF |
Source |
|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1639957 | PMC |
| http://dx.doi.org/10.1136/bmj.1.6018.1126 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Macrophage Activation Syndrome/Secondary Hemophagocytic Lymphohistiocytosis in Adult-Onset Still's Disease: An Uncommon Initial Presentation in a Young Nepalese Female: A Case Report.
Clin Case Rep
January 2025
Department of Rheumatology, Institute of Medicine Tribhuvan University Maharajgunj Kathmandu Nepal.
Hemophagocytic lymphohistiocytosis (HLH), is a fatal systemic hyperinflammatory syndrome. HLH may be due to immunosuppression, infections, cancer, or autoimmune diseases with fever and cytopenia. HLH which occurs in adult-onset Stills disease (AOSD) is called secondary HLH, also known as macrophage activation syndrome (MAS).
View Article and Find Full Text PDFDesigning a single-arm phase 2 clinical trial of mitapivat for adult patients with erythrocyte membranopathies (SATISFY): a framework for interventional trials in rare anaemias - pilot study protocol.
BMJ Open
July 2024
Institute of Health Policy, Management and Evaluation, Dalla Lana School of Public Health, University of Toronto, Toronto, Ontario, Canada.
Article Synopsis
- Membranopathies include blood disorders caused by genetic changes in red blood cell membrane proteins, with hereditary spherocytosis and stomatocytosis being key examples, while CDA II shows similar symptoms.
- Mitapivat, a new drug that activates pyruvate kinase, has shown promise in raising hemoglobin levels and reducing blood cell breakdown in various diseases and is being tested in a trial with around 25 adults who have membranopathies or CDA II.
- The SATISFY trial aims to determine the safety and effectiveness of mitapivat over an 8-week period, with a focus on measuring its impact on hemoglobin, hemolysis, and patient-reported outcomes.
Rare congenital Dyserythropoietic anemia of childhood: A case report.
Clin Case Rep
February 2023
Department of Nursing Hamad Medical Corporation Doha Qatar.
Congenital dyserythropoietic anemias (CDA) is a heterogeneous class of anemia of varying degrees of ineffective erythropoiesis and secondary hemochromatosis. We reported a case of CDA and showed our approach to reaching a diagnosis, highlighting the importance of the typical morphological appearance of bone marrow erythroblasts to reach the diagnosis.
View Article and Find Full Text PDF[A case of cholelithiasis that seems like secondary hemochromatosis as a result of congenital dyserythropoietic anemia].
Zhonghua Gan Zang Bing Za Zhi
August 2022
Senior Department of Hepatology, the Fifth Medical Center of PLA General Hospital, Beijing 100039, China.
Hematopoietic Cell Transplantation for Congenital Dyserythropoietic Anemia: A Report from the Pediatric Transplant and Cellular Therapy Consortium.
Transplant Cell Ther
June 2022
Division of Hematology, Oncology, Blood and Marrow Transplant, Nationwide Children's Hospital, Columbus, Ohio.
Hematopoietic cell transplantation (HCT) is the sole curative option for congenital dyserythropoietic anemia (CDA), a rare type of hemolytic anemia characterized by anemia, ineffective erythropoiesis, and secondary hemochromatosis. In this retrospective multicenter study, we report the outcomes of children with CDA who underwent HCT at participating Pediatric Transplantation and Cellular Therapy Consortium centers. Clinical information on HCT and associated outcomes was collected retrospectively using a common questionnaire.
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!