148: Long-Term Neurocognitive Function of Pediatric Patients with Severe Combined Immune Deficiency (SCID): Pre and Post Hematopoietic Stem Cell Transplant (HSCT)

M. Lin, K. Epport, C. Azen, A.J. Shah
2008 Biology of Blood and Marrow Transplantation  
recommendation. This includes patients who received only one of a recommended tandem autologous HSCT, or who received an autologous rather than a recommended allogeneic procedure. But given the limitations of this analysis, we conclude that restrictions imposed by health insurance coverage lead to significant delays in implementing the treatment plan for patients who receive HSCT. Significance: Chronic graft-versus-host disease (cGVHD) is an autoimmune-like reaction that causes debilitating
more » ... toms following allogeneic hematopoietic stem cell transplantation (al-loHSCT). The major barrier to good management of these symptoms is inadequate assessment. Theoretical Framework: Symptom burden is the combined impact of all symptoms on a person's ability to function as one did prior to onset of disease and therapy. Problem and Purpose: There is scant literature addressing the symptom burden of cGVHD. Symptoms are subjective phenomena and can only be accurately measured by patient report. The purposes of this research are to develop a short, easily understood, valid, and reliable patient questionnaire for measuring cGVHD symptom burden and to describe the symptom burden of cGHVD. This abstract reports preliminary results. Methods: The 13 core symptom items and 6 interference items of the M. D. Anderson Symptom Inventory (MDASI), rated on a scale of 0 to 10, are the base for the MDASI-GVH. In addition, 14 cGVHD-specific symptom items were generated from interviews with patients and the ratings of an expert panel. 128 patients with cGVHD and 64 patients post al-loHSCT without cGVHD will complete the MDASI-GVH. Information on performance status, quality of life, and a repeat MDASI will be collected on patients with cGVHD. Psychometric analyses will reduce the number of items to the optimal set describing symptom burden, determine predictive and discriminant validity, and establish internal consistency and test-retest reliability. The symptom burden of cGVHD will be described through descriptive, correlational, cluster, and factor analyses. Results: To date, 31 patients with cGVHD and 7 without have completed the MDASI-GVH. Average age is 49.3 years and 50% are male. Symptom severity and interference is displayed in Table 1 . Patients with cGVHD reported a unique cluster of severe symptoms that included skin problems, eye problems, muscle weakness, and joint stiffness. By February of 2008, we will report data on 45 patients with cGVHD and 20 patients without cGVHD. Implications: Patients with cGVHD tend to report more symptom severity and interference than patients post alloHSCT without cGVHD. The most severe symptoms patients with cGVHD report are cGVHD-specific. Further testing is ongoing, but the MDASI-GVH is an easily completed method of quantifying symptoms of cGVHD for clinician monitoring and use in trials testing methods to treat cGVHD.
doi:10.1016/j.bbmt.2007.12.157 fatcat:toespqrdwfag3hvp5cdkwnlk4q