The long-term and extensive efficacy of low dose thalidomide in a case of an untransfusable patient with Non-Transfusion-Dependent Thalassemia

Patients with Non-Transfusion-Dependent Thalassemia may require regular transfusion therapy. However, these patients are at risk of developing irregular antibodies, making them untransfusable. Second line treatment usually includes hydroxyurea, which however is not effective in all patients. Other t...

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Published in:Blood cells, molecules, & diseases molecules, & diseases, 2016-03, Vol.57, p.97-99
Main Authors: Ricchi, Paolo, Costantini, Silvia, Spasiano, Anna, De Dominicis, Gianfranco, Di Matola, Tiziana, Cinque, Patrizia, Ammirabile, Massimiliano, Marsella, Maria, Filosa, Aldo
Format: Article
Language:English
Subjects:
Antisickling Agents - adverse effects
Blood Transfusion
Bone marrow
Bone Marrow - drug effects
Bone Marrow - pathology
Drug Administration Schedule
Female
Fetal Hemoglobin - metabolism
Hemoglobin A2 - metabolism
Humans
Hydroxyurea - adverse effects
Immunosuppressive Agents - therapeutic use
Isoantibodies - biosynthesis
Macrophages
Middle Aged
NTDT
Splenectomy
Thalassemia - blood
Thalassemia - pathology
Thalassemia - surgery
Thalassemia - therapy
Thalidomide
Thalidomide - therapeutic use
Treatment Outcome
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Summary:Patients with Non-Transfusion-Dependent Thalassemia may require regular transfusion therapy. However, these patients are at risk of developing irregular antibodies, making them untransfusable. Second line treatment usually includes hydroxyurea, which however is not effective in all patients. Other treatment options include thalidomide, which has been reported to be safe and effective in selected patients. We report the case of a patient who experienced improvement of hemoglobin levels and of a part of NTDT related complications, following 36months of continuous therapy with low doses of thalidomide.
ISSN:1079-9796
1096-0961
DOI:10.1016/j.bcmd.2016.01.003