The purpose of this study is to assess the tolerance and efficacy of mobilizing hematopoietic stem cells after a single injection of plerixafor (0.24mg/kg) in 3 adult patients affected by sickle cell disease.
More information: clinicaltrials.gov, ITHANET Clinical Trials
The purpose of this Phase II dose-ranging study is to investigate pharmacokinetic (PK) and pharmacodynamic (PD) properties of various doses of ticagrelor followed by 4 weeks of twice-daily treatment in paediatric patients with sickle cell disease.
More information: clinicaltrials.gov, ITHANET Clinical Trials
Allo-hematopoietic stem cell transplantation(HSCT) is the only way to cure β-thalassemia major at present. To expand donor pool，the researchers of this study developed a haplo-identical HSCT (Hi-HSCT) platform. But in prior Hi-HSCT using high dose post-transplant Cyclophosphamide in patients with leukemia, cytopenia post-transplant often developed, which was considered as a symptom of GVHD. Therefore, the investigators add unrelated umbilical cord blood (UCB) to the Hi-HSCT. It has reported that, as third-party cells, UCB will reduce GVHD. The purpose of this study is to determine whether unrelated UCB following Hi-HSCT can improve outcomes of Hi-HSCT in patients with β-thalassemia major.
More information: clinicaltrials.gov, ITHANET Clinical Trials
This study aims to assess the new film-coated tablet formulation to the currently approved dispersible tablet formulation with regards to overall safety, Gastrointestinal (GI) tolerability, palatability, satisfaction and compliance.
More information: clinicaltrials.gov, ITHANET Clinical Trials
8th of May marks the international thalassaemia day aiming to raise awareness of the disease. On this special day, every year, Thalassaemia International Federation (TIF) focuses on a different theme that intervenes with the quality and appropriate treatment of patients and impacts their quality of life. This year's theme highlights the need to support policy decisions that reduce health inequalities, with particular focus on countries affected by the financial and economic crisis. The global financial crisis has already had wide-reaching social, economic, and political impact, with some of its most devastating consequences affecting the quality of the health care.
For more information visit the dedicated International Thalassaemia Day at the TIF website and the dedicated 8th of May blog and read the TIF's message. You can also watch the two videos below.