Special Issue "Sickle Cell Anemia: From Genetic Epidemiology to New Therapeutic Strategies"
Deadline for manuscript submissions: 25 August 2019
Sickle cell anemia is a monogenic disease associated with recurrent painful episodes, inflammation, hemolytic anemia, and progressive multiorgan damage. However, its clinical expression is highly variable and dependent on other genes. The most significant genetic factors associated with its severity are the HbF levels, alpha gene number, and beta-haplotypes. The polymerization of deoxygenated HbS is responsible for red cell sickling, causing impaired blood rheology, intra- and extravascular hemolysis and vaso-occlusion. Hemolytic anemia is associated with cerebral macrovasculopathy, priapism, leg ulcers, and pulmonary hypertension. Blood transfusions remain the mainstay of therapy for all severe crises and the reference treatment for stroke prevention. Hydroxyurea has been the first drug treatment able to reduce the rate of vaso-occlusive crises and acute chest syndromes. New molecules have emerged, such as crizanlizumab, an antibody against the P-selectin adhesion molecule that decreases the crisis rate, or voxelotor (GBT-440), a modulator of hemoglobin oxygen affinity that inhibits polymerization and improves baseline anemia. However, to date, hematopoietic stem cell transplantation is the only disease-reversing treatment modality and highly successful results obtained with matched-sibling donors support using other donors, such as haplo-identical and unrelated donors, and exploring autologous transplantation modified by gene-therapy.
The present Special Issue aims to describe the physiopathology of this complex disease and to specify the indications and the best period of life to prescribe these different therapeutics in patients with sickle cell anemia.
Dr. Francoise Bernaudin
Manuscript Submission Information
Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All papers will be peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.
Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Journal of Clinical Medicine is an international peer-reviewed open access monthly journal published by MDPI.
Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 1800 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.
- Sickle cell anemia
- Genetic epidemiology
- Stem cell transplantation (matched-sibling, haploidentical, unrelated, gene therapy)
- Cerebral vasculopathy
- New therapeutic molecules
- Kidney in sickle cell anemia
- Pulmonary hypertension and heart involvement