Abstract

This investigator is a pediatric hematologist whose long-term objective is to become an independent clinical investigator in health outcomes in sickle cell disease with a primary focus on the study of health-related quality of life (QOL). The overall goal of this project is, not only to understand the impact of common disease complications on the health status and health-related QOL of the child, but also to increase the applicability of health-related QOL instruments in children with sickle cell disease. Under the mentorship of well established investigators in the fields of hematology, sickle cell disease, and health-related QOL, the candidate proposes a tailored career development program that addresses the long-term objective above. The proposed didactic coursework will provide training in theory and fundamental skills specific to the candidate's short-term goals to 1) increase content knowledge in the QOL research field, 2) increase pertinent methodological skills related to QOL research and the study of health outcomes in sickle cell disease, 3) effectively communicate research ideas and findings to colleagues, patients, families and lay public, and 4) effectively manage the candidate's research career. This application proposes to validate a commonly used generic measure of health-related QOL and compare it to another widely used generic measure. In addition, the health-related QOL of children with overt stroke will be examined and compared to the health-related QOL of those with silent stroke and no cerebral infarction. The impact of family factors on the health-related QOL of the child will be determined. The assessment of health-related quality of life for these children will provide a critically important tool for the expanded assessment of treatment outcomes and contribute to a refined determination of the utilities of currently used therapies such as hydroxyurea and new, high-risk experimental treatment, such as alternative donor bone marrow transplantation.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Mentored Patient-Oriented Research Career Development Award (K23)
Project #
5K23HL080092-05
Application #
7682552
Study Section
Special Emphasis Panel (ZHL1-CSR-Q (F3))
Program Officer
Werner, Ellen
Project Start
2005-09-28
Project End
2011-08-31
Budget Start
2009-09-01
Budget End
2011-08-31
Support Year
5
Fiscal Year
2009
Total Cost
$126,604
Indirect Cost

  Institution

Name
Medical College of Wisconsin
Department
Pediatrics
Type
Schools of Medicine
DUNS #
937639060
City
Milwaukee
State
WI
Country
United States
Zip Code
53226

  Publications

Beverung, Lauren M; Strouse, John J; Hulbert, Monica L et al. (2015) Health-related quality of life in children with sickle cell anemia: impact of blood transfusion therapy. Am J Hematol 90:139-43
Panepinto, J A; Owens, P L; Mosso, A L et al. (2012) Concentration of hospital care for acute sickle cell disease-related visits. Pediatr Blood Cancer 59:685-9
Brandow, Amanda M; Weisman, Steven J; Panepinto, Julie A (2011) The impact of a multidisciplinary pain management model on sickle cell disease pain hospitalizations. Pediatr Blood Cancer 56:789-93
Brandow, Amanda M; Panepinto, Julie A (2011) Monitoring toxicity, impact, and adherence of hydroxyurea in children with sickle cell disease. Am J Hematol 86:804-6
Thornburg, Courtney D; Calatroni, Agustin; Panepinto, Julie A (2011) Differences in health-related quality of life in children with sickle cell disease receiving hydroxyurea. J Pediatr Hematol Oncol 33:251-4
Brandow, Amanda M; Panepinto, Julie A (2010) Hydroxyurea use in sickle cell disease: the battle with low prescription rates, poor patient compliance and fears of toxicities. Expert Rev Hematol 3:255-60
Brandow, Amanda M; Brousseau, David C; Pajewski, Nicholas M et al. (2010) Vaso-occlusive painful events in sickle cell disease: impact on child well-being. Pediatr Blood Cancer 54:92-7
Panepinto, Julie A; Hoffmann, Raymond G; Pajewski, Nicholas M (2010) The effect of parental mental health on proxy reports of health-related quality of life in children with sickle cell disease. Pediatr Blood Cancer 55:714-21
Brandow, Amanda M; Jirovec, Danielle L; Panepinto, Julie A (2010) Hydroxyurea in children with sickle cell disease: practice patterns and barriers to utilization. Am J Hematol 85:611-3
Brandow, Amanda M; Brousseau, David C; Panepinto, Julie A (2009) Postdischarge pain, functional limitations and impact on caregivers of children with sickle cell disease treated for painful events. Br J Haematol 144:782-8

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