Abstract
Although most common in tropical regions, population migration has meant that sickle cell disease is now one of the most prevalent genetic diseases worldwide. The issues and challenges faced by physicians and patients have been discussed by an international group of experts representing 4 key regions: the USA, Europe, Latin America, and the Middle East/Africa. Conclusive evidence to support the use of transfusion therapy for the prevention of stroke has resulted in key changes to patient management in all regions, and increasing numbers of patients are benefiting from this management approach. However, it is apparent that transfusion therapy is still under-utilized, largely due to concerns over iron overload, alloimmunization, limited blood supplies, and, sometimes, due to parental refusal. Once transfused, assessment and management of body iron levels can be poor, particularly in patients who are intermittently transfused. Compliance with chelation therapy regimens is a significant challenge, but new therapeutic options are likely to overcome some of the current barriers. Key requirements in all regions were considered to be the following: to provide greater physician, patient, and family education; to ensure effective transition from pediatric to adult care; and to establish national guidelines in order to ensure best practice is consistently applied.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 124-133 |
| Number of pages | 10 |
| Journal | Pediatric Hematology and Oncology |
| Volume | 28 |
| Issue number | 2 |
| DOIs | |
| State | Published - Mar 2011 |
Funding
Received and accepted September 2006. The SCD global experts and regional advisory board meeting in Sept. 2006, and delegate attendance, were supported by Novartis Pharmaceuticals Corporation. Financial support for medical editorial assistance was provided by Novartis Pharmaceuticals. The authors thank Ruth Tidey, PhD, for medical editorial assistance with the manuscript. Address correspondence to Elliott Vichinsky, MD, Medical Director, Hematology/Oncology Department, Children’s Hospital & Research Center Oakland, 747 52nd Street, Oakland, CA 94609, USA. E-mail: [email protected] E. Vichinsky has been a clinical investigator for Novartis and has received funds to present research at educational meetings. S. L. Thein is a member of the Exjade Speakers’ Bureau. J. Kwiatkowski has been a Consultant for Novartis in the past. P. Swerdlow has been consulting on study design and education on iron overload and receives research funding from Novartis for the 109 study and its extension study. J. Porter is on an Advisory Board, Speakers’ Bureau and receives research funding from Novartis. The authors alone are responsible for the content and writing of the paper.
Keywords
- iron chelation
- iron overload
- sickle cell disease
- stroke
- transfusion
ASJC Scopus subject areas
- Pediatrics, Perinatology, and Child Health
- Hematology
- Oncology
