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Outcomes in Children with Severe Vasculopathy from Sickle Cell Anemia after Treatment with Chronic Transfusion, Surgical Revascularization and/or Allogenic Hematopoetic Stem Cell Transplantation
Conclusions: The risk for cerebral infarction and/or vasculopathy progression after initiation of treatment with either chronic transfusion, HSCT or EDAS is still a major concern. Our data suggest HSCT and surgical revascularization with chronic transfusion provide the greatest benefit in reducing stroke risk and HSCT reduces risk for progression of a severe vasculopathy. Additional, large population studies are needed to clarify the risk.DisclosuresMajumdar: NIMHD: Research Funding. Campbell: Functional Fluitics: Membership on an entity's Board of Directors or advisory committees; Global Blood Therapeutics: Membership on ...
Source: Blood - November 21, 2018 Category: Hematology Authors: Johnson, C. W., Majumdar, S., Campbell, A. D., Magge, S., Darbari, D. S., Webb, J., Diab, Y. A., Nickel, R. S., Speller-Brown, B., Carpenter, J., Abraham, A. Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Poster I Source Type: research

Enhanced Long-Term Brain Magnetic Resonance Imaging Evaluation of Children with Sickle Cell Disease after Hematopoietic Cell Transplantation
Sickle cell disease (SCD) is the most common cause of stroke in children. Strokes and “clinically silent” infarcts are caused by progressive sickle vasculopathy in large and small vessels [1-4], leading to SCD brain vasculopathy. Both stroke types can affect neurological and cognitive function starting early in childhood [1,2,5-9]. Stroke or stroke risk is a frequent indication f or pediatric SCD transplantation [10-12]. Based on several studies using brain magnetic resonance imaging (MRI) and magnetic resonance angiography (MRA), hematopoietic stem cell transplantation (HCT) is believed to halt progression of vasculopathy [10,12-16].
Source: Biology of Blood and Marrow Transplantation - January 8, 2017 Category: Hematology Authors: Nancy S. Green, Monica Bhatia, Erica Y. Griffith, Mahvish Qureshi, Courtney Briamonte, Mirko Savone, Stephen Sands, Margaret T. Lee, Angela Lignelli, Adam M. Brickman Source Type: research

How I manage cerebral vasculopathy in children with sickle cell disease
Summary Sickle cell disease induces specific brain alterations that involve both the macrocirculation and the microcirculation. The main overt neurovascular complications in children are infarctive stroke, transient ischaemic attack and cerebral haemorrhage. Silent cerebral infarction, cognitive dysfunction and recurrent headache are also common. Cerebrovascular disease selectively affects children with the HbSS or HbS‐β0 genotypes (i.e. sickle cell anaemia). The incidence of stroke peaks between 2 and 5 years of age (1·02/100 patient‐years) and increases with the severity of the anaemia. Most strokes can be prevent...
Source: British Journal of Haematology - May 5, 2015 Category: Hematology Authors: Valentine Brousse, Manoelle Kossorotoff, Mariane Montalembert Tags: State of the Art Review Source Type: research

Transcranial Doppler Screening Adherence Among Children with Sickle Cell Anemia Seen in the Emergency Department
Conclusion: Patients with SCA who present to the ED and are nonadherent to TCD screening guidelines are less likely to have had a recent hematology clinic visit. Therefore, the ED may be an important location for identifying patients lost to regular clinic follow-up in need of a TCD. An intervention that specifically targets this patient population will likely improve TCD screening rates and stroke prevention.Figure.DisclosuresNo relevant conflicts of interest to declare.
Source: Blood - November 21, 2018 Category: Hematology Authors: Weisman, J. K., Diamond, C., Kappa, S., Nickel, R. S. Tags: 903. Outcomes Research-Non-Malignant Hematology: Poster II Source Type: research

Organ function indications and potential improvements following curative therapy for sickle cell disease
Hematology Am Soc Hematol Educ Program. 2022 Dec 9;2022(1):277-282. doi: 10.1182/hematology.2022000372.ABSTRACTCurative therapies for sickle cell disease include allogeneic hematopoietic stem cell transplantation (HSCT) and gene-modified autologous stem cell transplantation. HSCT has been used for 30 years with success measured by engraftment, symptom control, graft-vs-host disease (GVHD) risk, organ toxicity, and immune reconstitution. While human leukocyte antigen-matched sibling donor (MSD) transplants have excellent outcomes, alternate donor transplants (unrelated/haploidentical) are just beginning to overcome GVHD and...
Source: Hematology ASH Education Program - December 9, 2022 Category: Hematology Authors: Monica L Hulbert Allison A King Shalini Shenoy Source Type: research

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