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Characterization of Sickling During Controlled Automated Deoxygenation with Oxygen Gradient Ektacytometry

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Published on: November 5, 2019

Severe sickle cell disease--pathophysiology and therapy.

George Buchanan¹, Elliott Vichinsky, Lakshmanan Krishnamurti

¹University of Texas Southwestern, Dallas, Texas, USA.

Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation

|October 13, 2009

Summary

Sickle cell disease (SCD) affects millions globally, with hematopoietic stem cell transplantation (HSCT) offering a cure but remaining underutilized due to risks. Research aims to improve SCD management and optimize outcomes through better understanding and advanced therapies.

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Characterization of Sickling During Controlled Automated Deoxygenation with Oxygen Gradient Ektacytometry

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Continuous Manual Exchange Transfusion for Patients with Sickle Cell Disease: An Efficient Method to Avoid Iron Overload

Continuous Manual Exchange Transfusion for Patients with Sickle Cell Disease: An Efficient Method to Avoid Iron Overload

Published on: March 14, 2017

Area of Science:

Hematology
Genetics
Transplantation Medicine

Background:

Sickle Cell Disease (SCD) impacts over 70,000 individuals in the US and many more worldwide, with significant birth rates in African countries.
Current conservative management of SCD incurs substantial lifelong medical and psychosocial costs.
Hematopoietic stem cell transplantation (HSCT) presents the only potential cure for SCD but is significantly underused.

Purpose of the Study:

To highlight the current state of sickle cell disease management and treatment options.
To address the underutilization of hematopoietic stem cell transplantation (HSCT) for SCD.
To emphasize the need for ongoing research in SCD pathophysiology and therapeutic advancements.

Main Methods:

Review of current literature on SCD management and HSCT.
Analysis of data from the Center for International Blood and Marrow Transplant Research (CIBMTR) regarding HSCT for SCD.
Discussion of emerging research in SCD pathophysiology and novel therapeutic approaches.

Main Results:

SCD affects a large population, with high incidence in certain regions like Nigeria.
HSCT is an effective cure for SCD but is performed in less than 500 cases annually according to CIBMTR data, due to associated risks.
Advances in understanding SCD and transplantation are improving patient care.

Conclusions:

Despite advances, HSCT for SCD remains underutilized due to significant risks of morbidity and mortality.
Continued research into SCD pathophysiology, predictive modeling, and therapeutic timing is crucial for optimizing patient outcomes.
Improving the safety and accessibility of HSCT and exploring novel therapies are key to enhancing care for both pediatric and adult SCD patients.