Physical Health Netherlands Project #63926

Better treatment of Sickle cell disease

by Stichting Erasmus MC Foundation

Summary

Every year, 300,000 babies are born worldwide with sickle cell disease (SCD) and over 30 million people have it. It causes chronic anemia, severe pain attacks, can damage organs and can be fatal. In poor countries, 75% of children with SCD, where a cure is not possible, die before their fifth birthday. Two main subtypes are defined, HbSS and HbSC. This project focusses on HbSC. This study, investigates if phlebotomy (drawing blood) can be an affordable treatment for this subtype.

$190,000
total goal

$189,950
remaining

1
donor

0
monthly donors

7
months

Challenge

Sickle cell disease (SCD) is a devastating hereditary disorder. SCD patients have chronic anemia and severe pain attacks. The disease can damage organs. As a result the life expectancy of patients in the Western World is 20-30 years shorter than that of the general population. In low-income countries 75% of patients die before the age of 5. The most common types of SCD are SCA and HbSC. This study investigates the effectiveness of a treatment for HbSC to reduce the occurrence of complications.

Solution

This study aims to assess the effectiveness of phlebotomy (drawing blood) as treatment for HbSC. Phlebotomy is generally considered safe, with low risks. If this study shows positive results, it could lead to implementing phlebotomy globally as a treatment for HbSC, especially in low-income regions, such as African countries, where HbSC is prevalent. Phlebotomy is a relatively inexpensive therapy that can be easily performed globally and will therefore help millions of patients.

Long-Term Impact

If this treatment is successful it can easily and cost effectively be applied to millions of patients all over the world. This treatment will improve their quality of life and life expectancy immensely. If effective this treatment reduces the occurrence of complications like painful episodes, acute chest syndrome (a severe lung condition), irreversible damage to the bones, or irreversible damage to the eyes.