Therapy preference and decision-making among patients with severe sickle cell anemia and their families

Abstract

Background: Patients with severe sickle cell anemia (SCA) may benefit from therapeutic intervention with hydroxyurea (HU), chronic red cell transfusion (CT), or stem cell transplantation (SCT). Determination of best treatment is complicated by the tradeoff between each treatment's risks and benefits and the lack of data comparing them to determine efficacy. We explored factors that influenced making decisions regarding interventions and examined the relations between treatment preference and health-related quality of life (HRQOL).

Methods: Children with severe SCA and their parents received brochures describing each treatment, discussed risk/benefits with a nurse-educator, and answered questions regarding HRQOL and the factors influencing treatment preference. Severe SCA was defined as >or=3 pain events requiring ER visits or hospitalizations within 12 months, >or=2 acute chest syndrome (ACS) events within 24 months, or a combination of the two.

Results: Thirty parents and 7 patients participated. HU was preferred by 21 parents and 4 children, CT by 5 parents and 1 child, and SCT by 3 parents and 1 child. One parent was undecided and one child preferred no treatment. Interviewees were most influenced by perceived efficacy and safety, but no factors differed significantly among treatment preference groups. HRQOL median scores (0-100 scale) for parents (56; range, 28-91) and children (61; range, 31-96) did not differ significantly among treatment preference groups.

Conclusions: Patients with severe SCA and their parents can identify their treatment preferences. Improved understanding of their preferences and decision-making process will aid in the design of future clinical trials and in medical decision-making.