Trend and treatment patterns of aplastic anemia in Korea, pure red cell aplasia and myelodysplastic syndrome in Korea: a nation-wide analysis.
Trend and treatment patterns of aplastic anemia in Korea, pure red cell aplasia and myelodysplastic syndrome in Korea: a nation-wide analysis. Int J Hematol. 2017 Jun 29;: Authors: Choi Y, Jo JC, Jeon HJ, Kim DW, Chang MH, Kim H Abstract Aplastic anemia (AA) and pure red cell aplasia (PRCA) appear to be more prevalent in Asian countries including Korea. However, there are no exact data regarding its prevalence and frequency of allogeneic hematopoietic cell transplantation (HCT) in Korea. Here, we present demographic data relating to AA/PRCA/MDS in Korea. Data were prepared by retrieval from a computerized database maintained by the National Health Insurance Service and Korea National Statistical Office. HCT data were collected from all HCT centers in Korea. The crude incidence rate of AA decreased from 2002 to 2010 and from 35 to 28 per million persons. Females were more affected by AA. The peak ages of onset of AA were in the seventh decade or older. The frequency of HCT for AA increased from 2002 to 2012 and from 69 to 131 per year. The crude incidence rates of MDS increased from 2002 to 2010, with 8-20 per million persons, and the frequency of HCT also increased, from 30 in 2002 to 132 in 2011. Even allowing for the possibility of overestimation, the crude incidence of AA is significantly higher in Korea than in western countries. PMID: 28664500 [PubMed - as supplied by publisher]
Gamida Cell said today that the first patient has been transplanted in a study of its CordIn product for patients with severe aplastic anemia or hypoplastic myelodysplastic syndrome. The product is designed for patients with rare genetic diseases who have no fully-matched donors for a bone marrow transplantation. Gamida Cell said it is also evaluating its CordIn therapy for patients with sickle cell disease. Get the full story at our sister site, Drug Delivery Business News. The post Gamida Cell launches severe aplastic anemia trial appeared first on MassDevice.
This investigator initiated study is being conducted in collaboration with the U.S. National Heart, Lung and Blood Institute (NHLBI) JERUSALEM, Aug. 21, 2017 -- (Healthcare Sales &Marketing Network) -- The first subject has been transplanted in an in... Biopharmaceuticals Gamida Cell, Cordin, aplastic anemia, myelodysplastic syndrome
AbstractSince its discovery, the thrombopoietin (TPO) pathway has been an important pharmaceutical target for the treatment of thrombocytopenia. The first generation of TPO mimetics included peptide agents sharing homology with endogenous TPO, but these introduced a risk of antibody formation to endogenous TPO and were not successful. However, second-generation TPO mimetics or TPO receptor agonists (RAs) are currently being used to treat thrombocytopenia associated with a number of conditions, such as immune thrombocytopenia (ITP), severe aplastic anaemia (SAA), and hepatitis C virus-associated chronic liver disease. Accum...
• Recombinant human thrombopoietin (rhTPO) promotes platelet engraftment in patients after allogeneic hematopoietic stem cell transplantation;• The 3-year OS rate of patients with poor platelet graft function (PPGF) was significantly less than that of patients with good platelet graft function ( GPGF);• In patients with myelodysplastic syndromes (MDS) and aplastic anemia (AA), rhTPO was associated with a significant survival advantage.
Conclusion SDF-1/CXCR4 axis plays a crucial role in engraftment; however, more studies are warranted to assess their expression post-transplant. Evaluating the ligand (chemokine, SDF-1) or its receptor (CXCR4) may serve as potential surrogate markers for assessment of engraftment.
Authors: Shimamura A Abstract Clonal progression to myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) remains a dreaded complication for a subset of patients with bone marrow failure (BMF). Recognizing risk factors for the development of MDS or AML would inform individualized treatment decisions and identify patients who may benefit from early or upfront hematopoietic stem cell transplantation. Now that next-generation DNA sequencing is available in the clinical laboratory, research has focused on the implications of germ line and somatic mutations for diagnosing and monitoring patients with BMF. Most ...
Authors: Hasle H Abstract Myelodysplastic syndrome (MDS) and myeloproliferative disorders are rare in children; they are divided into low-grade MDS (refractory cytopenia of childhood [RCC]), advanced MDS (refractory anemia with excess blasts in transformation), and juvenile myelomonocytic leukemia (JMML), each with different characteristics and management strategies. Underlying genetic predisposition is recognized in an increasing number of patients. Germ line GATA2 mutation is found in 70% of adolescents with MDS and monosomy 7. It is challenging to distinguish RCC from aplastic anemia, inherited bone marrow failu...
This study shows that while any single specific bone marrow failure/myelodysplastic syndrome genetic disorder is rare, screening for these disorders in aggregate identifies a significant subset of patients with inherited bone marrow failure/myelodysplastic syndrome. PMID: 27418648 [PubMed - in process]
In conclusion, alloHCT from haploFD in sAA was comparable with alloHCT from AD, but extensive chronic GvHD seemed frequent in haploFD. Therefore alloHCT from haploFD could be an alternative approach for alloHCT from AD in adult sAA. PMID: 27409595 [PubMed - as supplied by publisher]
This study shows that while any single specific bone marrow failure/myelodysplastic syndrome genetic disorder is rare, screening for these disorders in aggregate identifies a significant subset of patients with inherited bone marrow failure/myelodysplastic syndrome. PMID: 27418648 [PubMed - as supplied by publisher]