Life expectancy and risk factors for early death in patients with severe thalassemia syndromes in South India.

In spite of advances in chelation therapy and screening of blood, mortality associated with the most common life-threatening noncommunicable disease of children in India, transfusion-dependent thalassemia (TDT), remains poorly defined. This study aims at estimating death rates and mortality risk factors associated with TDT. The clinical records of 1087 patients from 5 thalassemia centers in India were retrospectively analyzed from 2011 to 2018. Median patient age was 8.5 years, with 107 patients older than 18 years; 656 patients were male and 431 were female. Demographic details and clinical parameters were analyzed at presentation and at last visit. With 41 recorded deaths, actuarial survival at 26.9 years was 50%, and under-5 mortality was 7 times higher than in the general population. Patients with transfusion-transmitted infections (TTIs) had 3.4 times higher risk for death (P = .031). Serum ferritin higher than 4000 ng/dL had 4.6 times higher risk for mortality compared with ferritin lower than 1000 ng/dL (P = .00063). A hemoglobin drop lower than 2 g/dL per week had 7.7 times higher mortality risk compared with a drop of less than 1 g/dL per week (P < .0001). Social determinants (sex, economic status, and distance from center), splenectomy, and even cardiac complications were not associated with higher mortality risk. Main causes of death were infection, iron overload, TTIs, and allo-immunization. Patients who received more than 4 years of adequate care had more than 66% mortality risk reduction (P < .0001). TDT in India continues to result in high mortality. Ineffective transfusion, TTIs, and chelation continue to be the most significant risk factors. Comprehensive care in dedicated day care centers from early age is likely to improve outcomes.

Information Technology-Assisted Treatment Planning and Performance Assessment for Severe Thalassemia Care in Low- and Middle-Income Countries: Observational Study.

BACKGROUND: Successful models of information and communication technology (ICT) applied to cost-effective delivery of quality care in low- and middle-income countries (LMIC) are an increasing necessity. Severe thalassemia is one of the most common life-threatening noncommunicable diseases of children globally. OBJECTIVE: The aim was to study the impact of ICT on quality of care for severe thalassemia patients in LMIC. METHODS: A total of 1110 patients with severe thalassemia from five centers in India were followed over a 1-year period. The impact of consistent use of a Web-based platform designed to assist comprehensive management of severe thalassemia (ThalCare) on key indicators of quality of care such as minimum (pretransfusion) hemoglobin, serum ferritin, liver size, and spleen size were assessed. RESULTS: Overall improvements in initial hemoglobin, ferritin, and liver and spleen size were significant (P<.001 for each). For four centers, the improvement in mean pretransfusion hemoglobin level was statistically significant (P<.001). Four of five centers achieved reduction in mean ferritin levels, with two displaying a significant drop in ferritin (P=.004 and P<.001). One of the five centers did not record liver and spleen size on palpation, but of the remaining four centers, two witnessed a large drop in liver and spleen size (P<.01), one witnessed moderate drop (P=.05 for liver; P=.03 for spleen size), while the fourth witnessed a moderate increase in liver size (P=.08) and insignificant change in spleen size (P=.12). CONCLUSIONS: Implementation of computer-assisted treatment planning and performance assessment consistently and positively impacted indexes reflecting effective delivery of care to patients suffering from severe thalassemia in LMIC.