Therapeutic Intervention and Decision-Making by Parents of Children with Severe Sickle Cell Anemia.

Abstract Recurrent vaso-occlusive events in children with sickle cell anemia (SCA) prompt consideration of intervention with one of 3 treatments: hydroxyurea (HU), chronic red cell transfusion (CT) or stem cell transplant (SCT). Differing risks and benefits and a lack of randomized trials make it difficult to determine the best strategy. The preference of parents/caregivers (PC) is an important element in the decision process. To address these issues, we have completed a study with the following objectives: 1) To explore factors that influence PC decision-making (DM) regarding interventions, 2) To examine relationships between treatment preference and health-related quality of life (HRQOL). Severe SCA was defined as ≥3 pain events requiring ER visits or hospitalization within 12 months, or ≥2 acute chest syndrome events within 24 months, or a combination. PC were given literature in layman's language describing the treatments and later participated in a standardized discussion about treatment risks and benefits with an educator. PC then completed a 10-minute survey, which explored factors influencing treatment preference, and the HRQOL Peds QL 4.0 inventory. The survey had 40 questions grouped in different categories, including efficacy, safety, emotional factors, and practicability. HRQOL included physical, emotional, social and school functioning domains. Thirty PC were interviewed, 83% were female with a median age of 36 years (range 24–50); all were African-American. Twenty-two (73 %) PC felt they received enough information to make a choice and 26 (87%) thought their children needed treatment. Twenty-one (70%) PC expressed preference for HU, 3 (10%) for SCT, 5 (17%) for CT, one (3%) was undecided, and none preferred no treatment (p Motivation for Treatment Preference Factor* HU(%) SCT(%) CT(%) Safer 19(90) 2(67) 3(60) Fewer side effects 18(86) 3(100) 3(60) Less time commitment 5(24) 0 0 Less family impact 13(62) 3(100) 3(60) Concerns with disease severity 13(62) 2(67) 3(60) Concerns with death risk 9(43) 2(67) 3(60) “ Treatment is the best for my child” 16(76) 3(100) 2(40) Patient's fear 5(24) 2(67) 3(60) * p The HRQOL median scores, on a 0 to 100 scale, were 51 (range: 28–91), 66 (range: 64–90), and 56 (range: 53–77) among the HU, SCT, and CT groups respectively (p=0.2). We conclude that: HU was the most often preferred treatment option of PC of children with severe SCA. Efficacy and safety were the most common concerns influencing treatment preference, PC preferring SCT appear to have a distinct profile, with more concerns regarding disease severity and risk of death, greater endorsement of the “good parent concept”, and a perception of lower toxicity with the treatment. This may seem incongruent given the higher toxicity of SCT, but may reflect a perception of less long-term SCA-related complications (due to a possible cure). HU-preferring PC appear to have greater appreciation of treatment safety and time-commitment, and less concerns with the risk of death and patients' fears. There were no differences in HRQOL among the groups. Improved understanding of caregivers' preferences and their DM process will aid in the design of future clinical trials and medical decision-making.