Fred Hutch researcher gets $12.9M for 'bubble boy,' sickle-cell anemia work

Dr. Rainer Storb wants to make transplants safer and more widely available for patients who already suffer from immune diseases, such as "bubble-boy" disease. Now, Storb will have $12.9 million to work on that research, thanks to a grant from the National Heart, Lung, and Blood Institute. “Current approaches at cell and gene therapy for lethal noncancerous diseases of the blood and immune systems have inherent toxicities that may affect patients for the rest of their lives,” Storb said in a…
Source: Health Care:Hospitals headlines - Category: Hospital Management Authors: Source Type: news

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Chronic transfusion therapy with the goal of maintaining a hemoglobin (Hb) S
Source: Journal of Pediatric Hematology Oncology - Category: Hematology Tags: Clinical and Laboratory Observations Source Type: research
We report a case of MSI in a 12-year-old boy with homozygous sickle cell anemia (Hb SS) whose chronic transfusion therapy resulted in hypersplenism. The occurrence of a complicated MSI in our patient should perhaps further encourage elective splenectomy in such patients, despite known potential perioperative complications and postsplenectomy risks of infection and thrombosis.
Source: Journal of Pediatric Hematology Oncology - Category: Hematology Tags: Online Articles: Original Articles Source Type: research
Authors: Ogunsile FJ, Naik R, Lanzkron S Abstract INTRODUCTION: Venous thromboembolism (VTE) is a common comorbid condition found in sickle cell disease (SCD) and is associated with increased mortality for adults with SCD. The pathophysiology that leads to the thrombophilic state in SCD has been previously reviewed; however, evidence-based guidelines to aid in diagnosis, prevention and management of VTE are lacking. Areas covered: This review article will cover the pathophysiology underlying the hypercoagulable state, the epidemiology of VTE, and management strategies of VTE in SCD. Expert opinion: Providers should...
Source: Expert Review of Hematology - Category: Hematology Tags: Expert Rev Hematol Source Type: research
Daily administration of hydroxyurea is feasible and safe for children with sickle cell anemia in sub-Saharan Africa, according to newresearch.
Source: JAMA - Category: General Medicine Source Type: research
White matter integrity and processing speed in sickle cell anemia. Neurology 2018;90:e2042-e2050. Doi:10.11212/WNL.0000000000005644Stotesbury H, Kirkham FJ. Kolbel M, et al.
Source: Pediatric Neurology - Category: Neurology Authors: Source Type: research
Along with her research in sickle cell anemia, Wethers was also a trailblazer for African American women in the medical community.Medscape Medical News
Source: Medscape Medical News Headlines - Category: Consumer Health News Tags: Pediatrics News Source Type: news
CONCLUSION: The SO/DE protocol is an efficient, safe and cost-effective procedure for patients with sickle cell anemia under a chronic transfusion program. PMID: 30747440 [PubMed - as supplied by publisher]
Source: Transfusion - Category: Hematology Authors: Tags: Transfusion Source Type: research
Publication date: Available online 8 February 2019Source: Blood Cells, Molecules, and DiseasesAuthor(s): Obiageli Nnodu, Hezekiah Isa, Maxwell Nwegbu, Chinatu Ohiaeri, Samuel Adegoke, Reuben Chianumba, Ngozi Ugwu, Biobele Brown, John Olaniyi, Emmanuel Okocha, Juliet Lawson, Abdul-Aziz Hassan, Ijeoma Diaku-Akinwumi, Anazoeze Madu, Osita Ezenwosu, Yohanna Tanko, Umar Kangiwa, Ahmed Girei, Yetunde Israel-Aina, Adama LaduAbstractBackgroundSickle cell disease (SCD) is a neglected burden of growing importance.>312,000 births are affected annually by sickle cell anemia (SCA). Early interventions such as newborn screening, peni...
Source: Blood Cells, Molecules, and Diseases - Category: Hematology Source Type: research
Fernandez M, Piel FB, Adekile A Abstract BACKGROUND: Sickle cell disease (SCD) is a neglected burden of growing importance.>312,000 births are affected annually by sickle cell anaemia (SCA). Early interventions such as newborn screening, penicillin prophylaxis and hydroxyurea can substantially reduce the mortality and morbidity associated with SCD. Nevertheless, their implementation in African countries has been mostly limited to pilot projects. Recent development of low-cost point-of-care testing (POCT) devices for sickle haemoglobin (HbS) could greatly facilitate the diagnosis of those affected. METHODS:...
Source: Blood Cells, Molecules and Diseases - Category: Hematology Authors: Tags: Blood Cells Mol Dis Source Type: research
ConclusionIn SCA children, hyperferritinemia requiring iron chelation is most strongly related to blood transfusion. This situation concerned almost one in five children in present study; this shows the magnitude of the problem which is underestimated.
Source: BMC Hematology - Category: Hematology Source Type: research
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