Therapy preference and decision-making among patients with severe sickle cell anemia and their families
ABSTRACT 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).
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.
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.
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.
- SourceAvailable from: Ethel Jernigan
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- "In the past decade, more studies have been published describing overall HRQOL in children and adolescents with sickle cell disease (Hankins et al., 2007; Palermo, Schwartz, Drotar, & McGowan, 2002; Panepinto, O'Maher, DeBaun, Loberiza, & Scott, 2005; Panepinto, O'Maher, DeBaun, Rennie, & Scott, 2004; Panepinto, Pajewski, Foerster, Sabnis, & Hoffman, 2009; Stengenga, Ward-Smith, Hinds, Routhieaux, & Woods, 2004). We conducted a study to further assess HRQOL in these patients, with the primary hypothesis that HRQOL of children and adolescents with SCD was less than that of healthy children and adolescents. "
ABSTRACT: To assess health-related quality of life (HRQOL) in children and adolescents with sickle cell disease (SCD). The PedsQL 4.0 Generic Scales, a multidimensional self-report instrument that has been shown to be valid and reliable for use in children and adolescents with chronic illness, consists of 23 items that assess physical, emotional, social, and school functioning. Questionnaires were administered to 124 children and adolescents (ages 8 to 18 years, child self-report) with SCD (100 sickle cell anemia, 24 sickle β zero thalassemia) and their parents (parent-proxy report). Summary scores for children's and parents' ratings of overall HRQOL and psychosocial health and subscale scores for physical, emotional, social, and school functioning were compared with published data for healthy children. Both summary and subscale scores for children with SCD also were compared with those of their parents. Children with SCD and their parents rated overall HRQOL and all subdomains of HRQOL lower than did healthy children and their parents (P < .001). Children with SCD rated their own HRQOL significantly better than their parents did for overall HRQOL and all subdomains (P < .001) except emotional functioning (P = .06). Children with SCD and their parents perceived overall HRQOL and all HRQOL subdomains to be lower than scores reported in healthy children. Therefore, successful therapeutic efforts to improve HRQOL could represent important advances in the health of children with SCD.Journal of Pediatric Health Care 07/2011; 25(4):208-15. DOI:10.1016/j.pedhc.2009.12.006 · 1.44 Impact Factor
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ABSTRACT: Providers of care for children undergoing stem cell transplantation (SCT) skillfully combine the roles of scientist and clinician. As scientists, they apply scientific methods and disease theory in the creation and testing of new therapies and in the careful observation and exploration of treatment outcomes. As clinicians, they are capable of intuitively delivering care in a patient- and family-centered context of meaning and life values. The specialty of SCT has inherent aspects that make treatment decision making complex and potentially contentious. Having a strategy ready to implement in advance or at the time when treatment decisions need to be made will facilitate and enhance the decision making process for both the health care team and family members. Here we introduce the individualized care planning and coordination (ICPC) model as a practical approach to facilitate ethical and effective decision making in pediatric SCT settings. The ICPC is a 3-step model comprising (1) relationship--understanding the illness experience from the perspective of the patient and family, sharing relevant information, and assessing ongoing needs; (2) negotiation--prognosticating, establishing goals of care, and discussing treatment options; and (3) plan--generating a comprehensive plan of care that includes life and medical plans. Based on a foundation of a care of competence, empathy, compassion, communication, and quality, the ICPC model aims to diminish contentious family-staff interactions that can lead to mistrust and help guide treatment decision making. The ICPC model enhances communication among patients, families, and clinicians by revealing patient and family values and medical and quality-of-life priorities before reaching or even during critical decision points in the transplantation process.Biology of Blood and Marrow Transplantation 04/2007; 13(3):245-54. DOI:10.1016/j.bbmt.2006.11.013 · 3.40 Impact Factor
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ABSTRACT: Decision analysis is a simulation, model-based research technique in which investigators combine information from a variety of sources to create a mathematical model representing a clinical decision. This tool can be used to address many clinical dilemmas in pediatric hematology for which traditional clinical trials are unfeasible or impossible. This article outlines the basic steps of performing and analyzing a decision analysis tree and describes several decision analyses published in the field of pediatric hematology and how to evaluate and judge the decision analysis literature.Pediatric Clinics of North America 05/2008; 55(2):287-304, vii. DOI:10.1016/j.pcl.2008.01.004 · 2.12 Impact Factor