title: The long-term and extensive efficacy of low dose thalidomide in a case of an untransfusable patient with Non-Transfusion-Dependent Thalassemia.
abstract: 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.
authors.affiliation: UOSD Malattie rare del globulo rosso, AORN A. Cardarelli, Naples, Italy. Electronic address: pabloricchi@libero.it.
authors.forename: Paolo
authors.initials: P
authors.lastname: Ricchi
authors.affiliation: UOSD Malattie rare del globulo rosso, AORN A. Cardarelli, Naples, Italy.
authors.forename: Silvia
authors.initials: S
authors.lastname: Costantini
authors.affiliation: UOSD Malattie rare del globulo rosso, AORN A. Cardarelli, Naples, Italy.
authors.forename: Anna
authors.initials: A
authors.lastname: Spasiano
authors.affiliation: UOSC Anatomia Patologica, AORN A. Cardarelli, Naples, Italy.
authors.forename: Gianfranco
authors.initials: G
authors.lastname: De Dominicis
authors.affiliation: UOC Biochimica Clinica, AORN Ospedali Dei Colli, PO Monaldi, Naples, Italy.
authors.forename: Tiziana
authors.initials: T
authors.lastname: Di Matola
authors.affiliation: UOSD Malattie rare del globulo rosso, AORN A. Cardarelli, Naples, Italy.
authors.forename: Patrizia
authors.initials: P
authors.lastname: Cinque
authors.affiliation: UOSD Malattie rare del globulo rosso, AORN A. Cardarelli, Naples, Italy.
authors.forename: Massimiliano
authors.initials: M
authors.lastname: Ammirabile
authors.affiliation: UOSD Malattie rare del globulo rosso, AORN A. Cardarelli, Naples, Italy.
authors.forename: Maria
authors.initials: M
authors.lastname: Marsella
authors.affiliation: UOSD Malattie rare del globulo rosso, AORN A. Cardarelli, Naples, Italy.
authors.forename: Aldo
authors.initials: A
authors.lastname: Filosa
mesh_terms: D000986:Antisickling Agents / Q000009:adverse effects; D001803:Blood Transfusion; D001853:Bone Marrow / Q000187:drug effects / Q000473:pathology; D004334:Drug Administration Schedule; D005260:Female; D005319:Fetal Hemoglobin / Q000378:metabolism; D006443:Hemoglobin A2 / Q000378:metabolism; D006801:Humans; D006918:Hydroxyurea / Q000009:adverse effects; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007518:Isoantibodies / Q000096:biosynthesis; D008875:Middle Aged; D013156:Splenectomy; D013789:Thalassemia / Q000097:blood / Q000473:pathology / Q000601:surgery / Q000628:therapy*; D013792:Thalidomide / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D000986:Antisickling Agents; D007166:Immunosuppressive Agents; D007518:Isoantibodies; D013792:Thalidomide; D006443:Hemoglobin A2; D005319:Fetal Hemoglobin; D006918:Hydroxyurea
keywords: Bone marrow; Macrophages; NTDT; Thalidomide