Manisha Jain1, Prantar Chakrabarti2, Tuphan Kanti Dolai1, Pramit Ghosh3, Prakas Kumar Mandal1, Shuvra Neel Baul1, Rajib De4. 1. Department of Hematology, Nil Ratan Sircar Medical College and Hospital, Kolkata, India. 2. Department of Hematology, Vivekananda Institute of Medical Sciences, Kolkata, India. 3. Department of Community Medicine, Deben Mahata Government Medical College, Purulia, West Bengal, India. 4. Department of Hematology, Nil Ratan Sircar Medical College and Hospital, Kolkata, India. Electronic address: drrajibacademics@gmail.com.
Abstract
INTRODUCTION: Hemoglobin (Hb)-F inducers are known to improve Hb level and transfusion dependence in thalassemia. This pilot study was conducted to assess the efficacy and safety of Hb-F inducer thalidomide compared to hydroxyurea (HU) in Hb E-β thalassemia patients. METHODS: This was a prospective interventional single-centre study with 45 Hb E-beta thalassemia patients equally divided into group-I (thalidomide+folic acid), group-II (HU + folic acid) and group-III (folic acid). Response was assessed at various time intervals with 12-months follow up period. Primary end points were increment in Hb, Hb-F level and improvement in transfusion requirement; secondary end point were tolerability and safety. RESULTS: There was 100% responder (R: Hb-increment ≥1 g/dl) in group-I with 66.67% major responder (MaR: Hb-increment ≥2 g/dl), while there were 40% and 0% responder in group-II and III respectively. Hb-increment was significantly (p-value <0.0001) better in thalidomide arm compared to HU. The Hb-increment was attributable to both increase in Hb-F levels and reduction in ineffective erythropoiesis in thalidomide arm. Transfusion reduction was significantly better in group-I compared to group-II (100% vs 34%). No severe adverse effects was reported by patients of any group. CONCLUSION: Thalidomide showed a persistent significant Hb-increment and transfusion independence in Hb E-β thalassemia patients compared to HU.
INTRODUCTION: Hemoglobin (Hb)-F inducers are known to improve Hb level and transfusion dependence in thalassemia. This pilot study was conducted to assess the efficacy and safety of Hb-F inducer thalidomide compared to hydroxyurea (HU) in Hb E-β thalassemiapatients. METHODS: This was a prospective interventional single-centre study with 45 Hb E-beta thalassemiapatients equally divided into group-I (thalidomide+folic acid), group-II (HU + folic acid) and group-III (folic acid). Response was assessed at various time intervals with 12-months follow up period. Primary end points were increment in Hb, Hb-F level and improvement in transfusion requirement; secondary end point were tolerability and safety. RESULTS: There was 100% responder (R: Hb-increment ≥1 g/dl) in group-I with 66.67% major responder (MaR: Hb-increment ≥2 g/dl), while there were 40% and 0% responder in group-II and III respectively. Hb-increment was significantly (p-value <0.0001) better in thalidomide arm compared to HU. The Hb-increment was attributable to both increase in Hb-F levels and reduction in ineffective erythropoiesis in thalidomide arm. Transfusion reduction was significantly better in group-I compared to group-II (100% vs 34%). No severe adverse effects was reported by patients of any group. CONCLUSION:Thalidomide showed a persistent significant Hb-increment and transfusion independence in Hb E-β thalassemiapatients compared to HU.
Authors: Rayan Bou-Fakhredin; Lucia De Franceschi; Irene Motta; Maria Domenica Cappellini; Ali T Taher Journal: Pharmaceuticals (Basel) Date: 2022-06-16