PB2395 A TALE OF 9 CITIES: THALASSAEMIA IN GUANGXI PROVINCE, CHINA

Abstract

Background:At the invitation of two Chinese volunteer organizations, Dandelion and Sunflower Foundations, we visited YuLin, Beilu, Rongxian, Luchuan, Nanning, Guigang, Guiping (Twice), WuZhou and BaiSe during July and November 2018 and their surrounding regions From earlier acquired information development of services for thalassemia patients were in very early stages. Some children with thalassemia major are abandoned by their parents because they cannot afford to pay for the transfusions and the chelation therapy. Many of these children are then adopted to families in various western countries.Aims:The aim was to evaluate the children, suggest how treatment may be improved and where possible provide assistance so that the families may access all necessary treatment modalities so that they do not reach a state of despair and then abandon their child or children.Methods:An update on thalassemia was presented in English and translated to Chinese. Parents, who wished to have their children evaluated, were interviewed and their children were examined. In some centers written information was collected. In addition, some home visits were undertaken in some surrounding rural areas to evaluate a number of children, who had been identified by the Foundations. The information acquired are presented in the results. Clinical evaluations were performed. Verbal consent was obtained for anonymous publication of data and photographs. Because of time restraints and as evaluations had not always been formally arranged 157 patients In addition another 25 were briefly evaluatedResults:Data was recorded on 97 boys and 60 girls. Mutations on 93 were homozygous 17(5), 41‐42/17 (11),41‐42/IVS II 654(16), 41‐42/‐28(19),17/‐28(4),IVSII 654/‐28(1),homozygous 71‐72 (3), 71‐72/‐28 (1), HbE/Thal (2), HbH Constant Springs (12) and w with an SEA alpha mutation. Age was 4months to 14years(Mean 7.9yrs). Pre‐transfusion Hb range was 30‐90 g/L (Mean 66) and ferritin 135 to 13,000ng/ml (n = 120 mean 3700). Many children had significant frontal bossing, maxillary hyperplasia and gum margin hyperplasia. Spleen was often palpable at between 3‐6 cm below costal margin and in one case was 16 cm. Chelation was below recommended doses and very irregular. Deferasirox was taken by 20 (mostly Indian origin), 23 occasional deferroxamine, 24 deferiprone, 17 combination of latter two, 2 on deferasirox and deferiprone and 3 all three chelators. Two patients were receiving Chinese traditional medicine. Two patients were also taking Thalidomide in order to increase their hemoglobinSummary/Conclusion:The presentations seemed to empower the parents. It became clearly accepted that pre‐transfusion Hb levels should be between 90‐100 g/L. Advice on optimal chelation was given in every instance where indicated. Where necessary and feasible, resources for assistance or sponsorship to enable parents to pay for transfusion, chelation, bone marrow transplantation when safe and donor had been identified, were put into action. Even though we try to avoid splenectomy, it was recommended for a number of children. We encouraged the Foundations to extend their activities as much as possible throughout the provence. Overall, even though a relatively small number of patients were seen, this cross section of population with thalassemia reflects the province‐wide status and also some mechanisms by which assistance could be given to maximize outcomes.