title: Hydroxyurea therapy in paraparesis and cauda equina syndrome due to extramedullary haematopoiesis in thalassaemia: improvement of clinical and haematological parameters.
abstract: Patients with beta-globin disorders show amelioration of clinical condition by sustained synthesis of fetal haemoglobin in adult life. We report data on a patient with beta(o)-thalassaemia genotype and thalassaemia intermedia clinical phenotype. He received therapy with hydroxyurea (20 mg/kg/d) because of the presence of extramedullary masses causing paraparesis, neurogenic bladder and impotence. During therapy, the patient showed an improved clinical picture and a significant increase in total Hb (from 71.8 to 103.2 g/L) and a gamma/alpha globin synthetic ratio (from 0.39 to 0.68). The myelosuppressive effect of hydroxyurea was revealed by a decrease in CFU-GEMM, BFU-E, and CFU-GM. Therefore hydroxyurea can be effective in the treatment of patients with extramedullary haematopoiesis (EMH) who are not transfusion-dependent and cannot be treated with radiotherapy.
authors.affiliation: Department of Haematology, St. Eugenio Hospital, Rome, Italy.
authors.forename: P
authors.initials: P
authors.lastname: Cianciulli
authors.forename: T C
authors.initials: TC
authors.lastname: di Toritto
authors.forename: F
authors.initials: F
authors.lastname: Sorrentino
authors.forename: L
authors.initials: L
authors.lastname: Sergiacomi
authors.forename: A
authors.initials: A
authors.lastname: Massa
authors.forename: S
authors.initials: S
authors.lastname: Amadori
mesh_terms: D000328:Adult; D006411:Hematopoiesis, Extramedullary; D006801:Humans; D006918:Hydroxyurea / Q000627:therapeutic use*; D007558:Italy; D008297:Male; D020335:Paraparesis / Q000188:drug therapy* / Q000209:etiology; D011128:Polyradiculopathy / Q000188:drug therapy* / Q000209:etiology; D016896:Treatment Outcome; D017086:beta-Thalassemia / Q000150:complications*
chemical_list: D006918:Hydroxyurea