Clinicopathologic Grand Rounds: Clinical Cases from the NIH Clinical Center: Reversal of Chronic Complications of Sickle Cell Anemia with Hematopoietic Stem Cell Transplantation

Clinicopathologic Grand Rounds: Clinical Cases from the NIH Clinical Center: Reversal of Chronic Complications of Sickle Cell Anemia with Hematopoietic Stem Cell TransplantationPresented by: Lead Presenters: Nargues Weir, MD, Co-director, Research Development, NHLBI-Inova Advanced Lung Disease Program and Matthew Hsieh, MD, Staff Clinician, Molecular and Clinical Hematology Branch, NHLBI, NIHCategory: Clinical Center Grand RoundsAired date: 06/03/2015
Source: Videocast - All Events - Category: Journals (General) Tags: Past Events Source Type: video

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This study is par for the course, looking at Japanese Olympic participants. Interestingly, it hints at the upper end of the dose-response curve for physical activity, in that a longer career as a professional athlete may be detrimental in comparison to lesser degrees of exercise and training. From this large, retrospective cohort study targeting 3546 Japanese Olympic athletes, we observed significant lower mortality among Olympians compared with the Japanese general population. The overall standardised mortality ratio (SMR) was 0.29. The results were consistent with previous studies conducted in other non-Asian co...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
This study demonstrates for the first time that senescent cells secrete functional LTs, significantly contributing to the LTs pool known to cause or exacerbate idiopathic pulmonary fibrosis. Against Senolytics https://www.fightaging.org/archives/2019/11/against-senolytics/ There is no consensus in science that is so strong as to have no heretics. So here we have an interview with a naysayer on the matter of senolytic treatments, who argues that the loss of senescent cells in aged tissues will cause more harm to long-term health than the damage they will do by remaining. To be clear, I think this to be a ...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
Abstract Sickle cell disease afflicts millions of people worldwide and approximately 100,000 Americans. Complications are myriad and arise as a result of complex pathological pathways 'downstream' to a point mutation in DNA, and include red blood cell membrane damage, inflammation, chronic hemolytic anemia with episodic vaso-occlusion, ischemia and pain, and ultimately risk of cumulative organ damage with reduced patient lifespan. The National Heart, Lung, and Blood Institute's 2014 evidence-based guideline for sickle cell disease -management states additional research is needed before investigational curative the...
Source: Haematologica - Category: Hematology Authors: Tags: Haematologica Source Type: research
Discussion This case demonstrates successful cure of pre-B-ALL complicating XLA by alloSCT with restoration of B-cell development and functional antibody response. We are aware of only one previous case of pre-B-ALL in an XLA patient (21), which suggests that human BTK deficiency in itself does not predispose to pre-B-ALL. However, there are data to suggest that BTK may act as a tumor suppressor, and BTK deficiency may predispose to tumor development following a “second hit.” Mice with a genetic deficiency in Slp65, a gene encoding an adaptor protein that functions together with BTK, have a block in progenito...
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
Leukemia relapse occurring in donor cells, so called donor cell leukemia (DCL) after allogeneic hematopoietic stem cell transplantation has been previously reported in the literature. Some authors have suggested that the development of DCL is perhaps a more common occurrence than traditionally thought. Donor cell myeloma (DCM) seems to be less frequent than DCL. This 46-year old male when first seen in 2000 was diagnosed with stage IIIa multiple myeloma. A monoclonal IgA kappa spike was recorded at diagnosis. Treatment with melphalan and prednisone was delivered every four to six weeks for a total of 22 courses. Fourty mon...
Source: Blood - Category: Hematology Authors: Tags: 723. Clinical Allogeneic and Autologous Transplantation: Late Complications and Approaches to Disease Recurrence Source Type: research
Sickle cell anemia (SCA) is caused by a point mutation in the beta-globin gene. SCA has potentially devastating consequences including chronic hemolytic anemia, episodic vascular occlusion, inflammation and oxidative stress, and cumulative multi-organ damage resulting in early mortality. In fact, with reduction of childhood mortality due to early diagnosis and infection prophylaxis, end-organ damage is the major cause of death in SCA. SCA patients show hyper coagulative state in the absence of vascular occlusion. Recently, our group has shown that reduction of circulating major clotting factor, thrombin, in SCA mice signif...
Source: Blood - Category: Hematology Authors: Tags: 113. Hemoglobinopathies, Excluding Thalassemia-Basic and Translational Science: Sickle Cell Disease-Role of Coagulation and Inflammation in Pathophysiology Source Type: research
We report pulmonary function analyzed by spirometry, before and after transplantation, in a group of Nigerian SCA children. Our results indicate that restrictive pulmonary pattern (RVP) is a common finding in these patients. Although we do not observe significant improvements in lung function after 3-6 months of transplantation, it is useful to perform a spirometric evaluation of these patients at the time of transplant.
Source: Mediterranean Journal of Hematology and Infectious Diseases - Category: Hematology Source Type: research
Imagine: inside the veins of an African-American child, red blood cells: round and soft, doing their job, keeping the person alive. What would happen if those cells hardened and changed shape, curving into the letter "C", like a wheat-cutting sickle? First, the capillaries would clog, in what Sickle Cell Disease (SCD) doctors call a "crisis". Excruciating agony, like broken glass in the veins, a crisis may last an hour or a day, and the pain is just the beginning. "By twenty years of age, about 15% of children with SCD suffer major strokes...by 40 years of age, almost half have central nervous sys...
Source: Science - The Huffington Post - Category: Science Source Type: news
Investigators at the National Heart, Lung, and Blood Institute have designed a novel lentiviral vector as a potential gene therapy for sickle cell anemia and beta-thalassemia. The novel lentiviral vector encodes the beta-globin gene in a forward orientation and can produce 5-10 fold higher viral titer and 4-10 fold higher gene transfer efficiency to hematopoietic stem cells than reverse-oriented lentiviral vectors. In vivo studies conducted in rhesus macaques show beta-globin production after transplantation with this novel lentiviral vector. This technology could provide an alternative therapy for patients suffering from ...
Source: NIH OTT Licensing Opportunities - Category: Research Authors: Source Type: research
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