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Description of the First Cases with ADAMTS13 Mutations in Hungary
CONCLUSIONS:Clinical and molecular characterization of the first 5 Hungarian cases with ADAMTS13 mutations was presented. Compound heterozygous, damaging ADAMTS13 mutations with deficient plasma ADAMTS13 activity were associated with serious clinical consequences. In case of thrombotic microangiopathy in young patients with stroke, or HELLP syndrome, complete workup for ADAMTS13 activity and inhibitors is necessary, and genetic analysis should be considered. Three new ADAMTS13 mutations (c.1016_1017delCA, c.3199T>A , c.2839C>T) were identified and shown to be associated with decreased plasma ADAMTS13 activity.Disclos...
Source: Blood - November 21, 2018 Category: Hematology Authors: Reti, M., Sinkovits, G., Cseprekal, O., Csuka, D., Szilagyi, A., Farkas, Z., Klucsik, Z., Szederjesi, A., Wagner, L., Reusz, G., Kremer Hovinga, J. A., Rigo, J., Masszi, T., Prohaszka, Z. Tags: 311. Disorders of Platelet Number or Function Source Type: research

The Glomerulopathy of Sickle Cell Disease
Abstract Sickle cell disease (SCD) produces many structural and functional abnormalities in the kidney, including glomerular abnormalities. Albuminuria is the most common manifestation of glomerular damage, with a prevalence between 26% and 68% in adult patients. The pathophysiology of albuminuria in SCD is likely multifactorial, with contributions from hyperfiltration, glomerular hypertension, ischemia‐reperfusion injury, oxidative stress, decreased NO bioavailability and endothelial dysfunction. Although its natural history in SCD remains inadequately defined, albuminuria is associated with increased echocardiography�...
Source: American Journal of Hematology - May 1, 2014 Category: Hematology Authors: Kenneth I. Ataga, Vimal K. Derebail, David R. Archer Tags: Critical Review Source Type: research

Successful kidney transplantation in a patient with congenital thrombotic thrombocytopenic purpura (Upshaw-Schulman syndrome).
CONCLUSION: Our experience suggests that kidney transplantation may be an appropriate management for carefully selected patients with congenital TTP who develop end-stage renal disease. PMID: 28940540 [PubMed - as supplied by publisher]
Source: Transfusion - September 20, 2017 Category: Hematology Authors: Fattah H, Kumar D, George JN, Massey HD, King AL, Friedman KD, Gupta G Tags: Transfusion Source Type: research

Chronic organ failure in adult sickle cell disease.
Authors: Vichinsky E Abstract Sickle cell disease is now a chronic adult illness characterized by progressive multiorgan failure, particularly involving the brain and kidney. The etiology is multifactorial; it includes hemolysis and nitric oxide deficiency. As patients age, most experience neurologic insult. Twenty-five percent of older adults have had a clinical stroke and at least half of the population have had a silent infarct, cortical atrophy, and neurocognitive impairment. Periodic screening with neuroimaging and neurocognitive testing is recommended. Identification and correction of modifiable risk factors ...
Source: Hematology ASH Education Program - December 10, 2017 Category: Hematology Tags: Hematology Am Soc Hematol Educ Program Source Type: research

Medical Conditions Among Survivors of Adolescent and Young Adult Non-Hodgkin Lymphoma (NHL), Acute Lymphoblastic Leukemia (ALL) and Acute Myeloid Leukemia (AML)
Conclusion: This study found that sociodemographic factors were associated with the risk of developing medical conditions in AYA NHL, ALL and AML survivors. As expected, the risk of medical conditions varied by cancer type and treatment, with those undergoing SCT having a higher risk of medical conditions regardless of cancer type. NHL and ALL survivors who were uninsured or publicly insured were at a consistently higher risk of developing medical conditions, as were Hispanic ALL survivors and Black AML survivors. Our findings highlight the higher burden of medical conditions in subgroups of cancer survivors that may relat...
Source: Blood - November 21, 2018 Category: Hematology Authors: Keegan, T. H. M., Muffly, L. S., Li, Q., Alvarez, E., Brunson, A. M., Malogolowkin, M., Wun, T. Tags: 904. Outcomes Research-Malignant Conditions: Real World Outcomes Source Type: research

Efficacy Evaluation of Liver-Directed Gene Therapy in Fabry Mice
Conclusions: Collectively, these data provide strong evidence that our liver-directed AAV-mediated gene therapy approach holds considerable therapeutic potential for the treatment of Fabry disease. We anticipate that a single dose IV procedure will pose minimal burden to Fabry patients and will be a viable alternative to biweekly enzyme infusions, potentially reducing treatment-related morbidity whislt improving patient quality of life and potentially providing them with a functional long-term cure.DisclosuresKia: Freeline: Employment, Equity Ownership. McIntosh: Freeline: Consultancy. Hosseini: Freeline: Employment, Equit...
Source: Blood - November 21, 2018 Category: Hematology Authors: Kia, A., McIntosh, J., Rosales, C., Hosseini, P., Sheridan, R., Spiewak, J., Mills, K., Corbau, R., Nathwani, A. C. Tags: 801. Gene Therapy and Transfer: Poster I Source Type: research

Final Results from a Phase I Trial Combining Selinexor with High-Dose Cytarabine (HiDAC) and Mitoxantrone (Mito) for Remission Induction in Acute Myeloid Leukemia (AML)
Conclusions: The selinexor/HiDAC/Mito regimen is feasible and tolerable at selinexor doses up to 80mg/day or ~50 mg/m2/day twice weekly. This regimen yields an ORR of 64% based on currently available data. We had previously reported molecular correlatives demonstrating the effect of selinexor. The recommended phase 2 dose is 80mg of selinexor.Figure.DisclosuresLarson: Ariad/Takeda: Consultancy, Research Funding; BristolMyers Squibb: Consultancy, Research Funding; Novartis: Consultancy, Research Funding; Pfizer: Consultancy, Research Funding. Odenike: Agios: Research Funding; Astex: Research Funding; Dava Oncology: Consulta...
Source: Blood - November 21, 2018 Category: Hematology Authors: Wang, A., Weiner, H., Larson, R. A., Odenike, O., Artz, A. S., Bishop, M. R., Godley, L., Thirman, M., Kosuri, S., Churpek, J., Curran, E. K., Pettit, K., Stock, W., Liu, H. Tags: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster III Source Type: research

High molecular weight kininogen contributes to early mortality and kidney dysfunction in a mouse model of sickle cell disease.
CONCLUSIONS: We found elevated plasma levels of cleaved HK in sickle patients compared to healthy controls, suggesting ongoing HK activation in SCD. We used bone marrow transplantation to generate wild type and sickle cell mice on a HK-deficient background. We found that short-term HK deficiency attenuated thrombin generation and inflammation in sickle mice at steady state, which was independent of bradykinin signaling. Moreover, long-term HK deficiency attenuates kidney injury, reduces chronic inflammation, and ultimately improves of sickle mice. PMID: 32573897 [PubMed - as supplied by publisher]
Source: Thrombosis and Haemostasis - June 22, 2020 Category: Hematology Authors: Sparkenbaugh EM, Kasztan M, Henderson MW, Ellsworth P, Davis PR, Wilson KJ, Reeves B, Key NS, Strickland S, McCrae K, Pollock DM, Pawlinski R Tags: J Thromb Haemost Source Type: research