[ effect of hudroxyurea on extramedullary hematopoietic masses in patients with beta-thalassemia intermediate]

ABSTRACT

Since the need for transfusions in thalassemia intermediate [TI] patients is limited into some rare occasions they are almost always referred complaining of problems due to chronic anemia, bone marrow hyper-functioning [such as gall stones, osteoporosis] and also extramedullary hematopoiesis masses [EHM]. Different treatment modalities have been reported for managing the latter complication. We carried out this study to evaluating the effects of Hydroxyurea in reducing the size of such masses. The study was clinical trial comparing before and after characteristics of the patients referred to the Talassemia Research center from 2003 to 2006. Presentation of all was pain, weakness, paresthesia of legs and paraparesia/paraplegia. Diagnosis of EHM was confirmed by Spinal MRI [a 1.5 Tesla, Toshiba machine]. Hydroxiurea [Syrea, Medac, Germany] was administered 10-20 mg/kg/day. CBC, Urea, Creatinin were checked monthly. A Neurologist was observing the progression of patients. The second MRI was performed 3-6 months later. Another radiologist unaware of the sequence of imaging reported the changes of EHM during the period. Paired-t test and McNemar test were applied to compare the results. P< 0.05 was considered as statistically significant. Eight patients [7 males and 1 female] were enrolled in our study. Their age ranged from 26-44 years old. None had undergone transfusion, nor had they used pain killers. Neurologic symptoms were initiated from one week to a couple of months prior to their admission. They had administered Hydroxyurea in recommended dosage. The follow up takes 0.7 - 4.7 years. All subjects improved within 2 weeks, whereas none of them needed surgical procedures and radiotherapy. Both the MCV and NRBC changes were significant [P<0.02]. No considerable side-effects were noticed within the follow up period. HU is proved to be both effective and safe in treatment of EHM amongst patients with thalassemia intermediate who develop neurological symptoms. We suggest hydroxyurea as a "prophylactic agent" for several complications of TI