Abstract:
Background: b-thalassaemia is an inherited disorder of haemoglobin synthesis which results in severe transfusion-dependent anaemia from infancy. Although considered a life-limiting disease, it can be cured by allogeneic haematopoietic stem cell transplantation and gene therapy. However, many patients and their families in developing countries are unaware of these treatment options. Objectives: To assess the maternal knowledge on curative therapies and to determine its association with the adequacy of current medical treatment and psychological health among children with b-thalassaemia. Method: We conducted a cross-sectional study at the three largest thalassaemia centres of Sri Lanka. All patients with transfusion-dependent b-thalassaemia aged 2-18 years were eligible for the study. Data were collected using an interviewer-administered questionnaire by interviewing mothers and from medical records. The questionnaire contained questions to gather information on socio-demographic background, clinical details and maternal knowledge on curative therapies for thalassaemia. The psychological morbidity of children was assessed using the previously validated ‘strengths and difficulties questionnaire’. Binary logistic regression was used in the analysis. Results: A total of 304 patients (mean age 9.8years; females 54%) were recruited. A majority (86%) of mothers knew that b-thalassaemia can be cured by haematopoietic stem cell transplantation; however, only 1% were aware of gene therapy. Detailed knowledge on curative therapies was lacking in most mothers; only 22% could identify suitable donors for transplantation. Maternal knowledge on curative therapies was associated with higher educational level and income of parents. Accurate maternal knowledge on haematopoietic stem cell transplantation was significantly associated with lower rates of hepatomegaly, splenomegaly, emotional symptoms, conduct symptoms, hyperactive symptoms and abnormal peer relationships in patients. Conclusions: This study demonstrated that maternal knowledge on curative therapies among patients with b-thalassaemia is sub-optimal. It further demonstrated that having an accurate maternal knowledge is associated with improved medical care and a lower prevalence of psychological symptoms among patients.
