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Updated: Jun 15, 2025
Continuous Manual Exchange Transfusion for Patients with Sickle Cell Disease: An Efficient Method to Avoid Iron Overload
Published on: March 14, 2017
Position paper on advancing sickle cell disease management in France by bridging the clinical practices and
K Benmoussa1, F Bernaudin2, P Connes3
1Terumo Blood and Cell Technologies, Ikaroslaan 41, Zaventem 1930, Belgium.
Insights
Sickle cell disease (SCD) management in France involves expert insights on clinical practices and guideline alignment. Key opinion leaders discussed current treatments, including automated red blood cell exchange, to improve patient care pathways.
Area of Science:
- Hematology
- Genetics
- Rare Diseases
Background:
- Sickle cell disease (SCD) is the most common rare disease in France, affecting thousands and causing severe complications.
- The genetic disorder stems from a β-globin gene mutation, leading to abnormal hemoglobin (HbS), hemolytic anemia, and impaired red blood cell function.
- Patient care pathways for SCD in France require clarification and optimization.
Purpose of the Study:
- To gather insights into the management of sickle cell disease (SCD) in France.
- To align current clinical practices with existing French guidelines for SCD.
- To identify and address regional disparities in SCD care.
Main Methods:
- A roundtable advisory board meeting was convened in France.
- Key opinion leaders (KOLs) in SCD management, including clinicians and researchers, participated.
- Discussions focused on clinical practices, guideline concordance, regional disparities, and available prophylactic/therapeutic options.
Main Results:
- KOLs evaluated the alignment of current SCD clinical practices with French guidelines, noting areas of both agreement and disagreement.
- Disparities in SCD management across different French regions and medical centers were identified.
- Current prophylactic and therapeutic strategies, particularly automated red blood cell exchange (aRBCX), were discussed.
Conclusions:
- The advisory board meeting provided a valuable platform for expert perspectives on SCD management in France.
- Findings highlight the need for improved guideline adherence and potential contributions to future guideline updates.
- Addressing identified disparities is crucial for optimizing SCD patient care pathways.
Abstract:
In France, sickle cell disease (SCD) is the most common rare disease and represents the most prevalent genetic disorder, with 19,800 to 32,400 patients diagnosed in 2016 and 1:714 newborns affected in 2019. SCD is caused by a single mutation in the β-globin gene, resulting in the production of abnormal hemoglobin (called HbS), chronic hemolytic anemia, and impaired red blood cell rheology. SCD patients face several severe acute and chronic complications, including stroke, acute chest syndrome (ACS), painful vaso-occlusive crisis (VOC), organ failure, and a high risk of infections. As patients' care pathway remains unclear in France, a roundtable advisory board meeting was organized in the country to provide insights into the management of SCD in alignment with clinical guidelines. The meeting brought together a panel of esteemed key opinion leaders (KOLs) in SCD management, encompassing both clinical practice and research. During the meeting, the KOLs discussed clinical practices and their alignment with French guidelines, identifying areas of concordance and discrepancy. They also addressed disparities in SCD clinical practices across regions and medical centers. The KOLs discussed the prophylactic and therapeutic options currently available for SCD patients in France, with a focus on transfusion therapies, especially automated red blood cell exchange (aRBCX). The results of this advisory board meeting provide a valuable platform for gathering expert perspectives on SCD management, clinical practices, guideline alignment, and the potential for contributions to guideline updates.
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