How do we translate gaps and unmet needs of blood management for thalassemia into a collaborative implementation framework?

BACKGROUND AND OBJECTIVE: The mainstay of management for thalassemia is regular blood transfusions. However, gaps and unmet needs of blood services for thalassemia are still not clearly identified and addressed in Thailand, a country prevalent with thalassemia. What can be a collaborative implementation framework that helps advance practices and policies relating to blood management for thalassemia? METHODS: The first Blood & Beyond Roundtable Discussion was held in July 2022 to gather the current situation, gaps, and unmet needs of blood services for thalassemia from multidisciplinary experts and thalassemic patients. The Implementation Guide as suggested by the Centre for Effective Services was applied as a tool to consolidate information from the discussions and construct the collaborative implementation framework. RESULTS: The National Blood Center and hospitals in Thailand followed the missions specified in the National Blood Policy and the standard guidelines to ensure the best practice of blood management for thalassemia. However, there were six gaps and unmet needs identified from the discussions. After all discussion points were mapped onto the framework, an implementation plan comprised of five specific activities became clear and actionable. CONCLUSION: Without the complete information from both experts and patients, the implementation plan would not have been successfully constructed. The method that we employed to translate all information into the framework can be adapted by other countries to develop their own specific framework efficiently.

Outcomes of transplantation with related- and unrelated-donor stem cells in children with severe thalassemia.

Recently published reports indicate that the outcome of unrelated donor transplantations in patients with leukemia is currently comparable to that of transplantation from identical family donors. We investigated the possibly favorable outcomes of related and unrelated transplantation in children with severe thalassemia. We reviewed transplantation outcome in 49 consecutive children with severe thalassemia who underwent allogeneic stem cell transplantation with related-donor (n=28) and unrelated-donor (n=21) stem cells between September 1992 and May 2005 at the Faculty of Medicine, Ramathibodi Hospital, Mahidol University (Bangkok, Thailand). Analysis of engraftment, frequency of procedure-related complications, and thalassemia-free survival showed no advantage from use of related-donor stem cells. The 2-year thalassemia-free survival estimate for recipients of related-donor stem cells was 82% compared with 71% in the unrelated-donor stem cell group (P=.42). The present study provides evidence to support the view that it is quite reasonable to consider unrelated-donor stem cell transplantation an acceptable therapeutic approach in severe thalassemia, at least for patients who are not fully compliant with conventional treatment and do not yet show irreversible severe complications of iron overload.

Absence of factor V Arg306--Thr and low factor V Arg306->Gly mutation prevalence in Thai blood donors.

Thrombosis among the Thai population is much lower than in western countries. The Thai population is protected to some extent against familial thrombophilia as by the very low prevalence of factor V Leiden, G20210A prothrombin and C677T methylenetetrahydrofolate reductase mutations. The present study reports the prevalence of two mutations of the factor V gene involving the codon for Arg 306 among 500 healthy adult voluntary blood donors (males 285, females 215) and 30 children (boys 20, girls 10) experiencing a total of 36 thrombotic episodes. The blood donors' ages ranged from 18 to 60 years while the children's ages ranged from 9 months to 15 years. The allelic frequencies of the factor V gene mutation of G1091C and A1090G among blood donors were 0% and 0.4%, respectively. Additionally, both mutations were not present in any of the 30 children with thrombosis. The low prevalence of factor V gene mutations in the codon Arg 306 may be relevant to the low rate of thrombosis among the Thai population.