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In a proof-of-concept study, HQK-1001 at 10, 20, 30, and 40?mg/kg administered daily for 8?weeks in 21 subjects with non-transfusion dependent ��-thalassaemia was well-tolerated (Fuchareon et?al, 2013). HQK-1001 at 20 mg/kg, which provided the best results, increased HbF in eight of nine subjects with a median increase of 6��6% and 4��4?g/l, and increased http://www.selleckchem.com/products/Fludarabine(Fludara).html total haemoglobin in four of nine subjects by a mean of 11?g/l. Here, a single-centre study was conducted to evaluate HQK-1001 at 20?mg/kg per day administered for a longer period (NCT 01642758). Adult patients with ��-thalassaemia intermedia characterized by two ��-globin mutations were eligible if their haemoglobin was between 60 and 90?g/l on two occasions during https://en.wikipedia.org/wiki/Chlormezanone the 30-d screening period. Patients were excluded if they were transfused within the previous 3?months, received iron chelation agents within the previous 7?d, another investigational agent within the previous 30?d, erythropoietic agents within the previous 90?d, or hydroxycarbamide within the previous 6?months, or had pulmonary hypertension requiring oxygen therapy, alanine aminotransferase (ALT) > four times the upper limit of normal, or serum creatinine?>135?��mol/l. HQK-1001 capsules (HemaQuest Pharmaceuticals, San Diego, CA, USA) was administered at 20?mg/kg once daily for 24?weeks. Folic acid was given daily, and to prevent iron-deficient inefficient erythropoiesis, oral iron was given if serum ferritin was 2250?pmol/l. After signing an Ethics Committee approved informed consent form, subjects were assessed clinically and underwent laboratory tests twice during a 30-d screening period, every 4?weeks while receiving HQK-1001, and then 4?weeks after the end of dosing. Ten subjects were enrolled, seven male and three http://www.selleckchem.com/products/Nolvadex.html female, with a mean age of 29��4?years (range 18�C52?years). Eight subjects were splenectomized; two had palpable splenomegaly at 4 and 7?cm below the left costal margin. The mean (range) baseline values were: HbF 26��6% (7��9�C73��8%), absolute HbF 20��1?g/l (5��5�C53��9?g/l), total haemoglobin 77��4?g/l (61��5�C96��0?g/l), platelet count 782?��?109/l (486�C1039?��?109/l), reticulocytes 10��9% (7��1�C15��7%), and serum ferritin 3188?pmol/l (375�C9772?pmol/l). Nine subjects completed the study and one subject was discontinued at Week 16 because of worsening anaemia requiring a transfusion. Mean compliance with HQK-1001, calculated as the ratio of the number of HQK-1001 capsules taken divided by the number of capsules prescribed, was 92��5%; two subjects had compliance