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Hydroxyurea therapy in paraparesisand cauda equina syndrome due to extramedullary haematopoiesis in thalassaemia: improvement of clinicaland haematological parameters

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Abstract: Patients with β‐globin disorders show amelioration of clinical condition by sustained synthesis of fetal haemoglobin in adult life.

We report data on a patient with β°‐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 γ/α 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.
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Keywords: BFU‐E; extramedullary haematopoiesis; hydroxyurea; spinal cord compression β‐thalassaemia

Document Type: Case Report

Affiliations: 1: Department of Haematology and 2: Department of Radiology, St. Eugenio Hospital, Rome and 3: Istituto superiore di Sanità, Vegata University, Rome, Italy

Publication date: June 1, 2000

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