A Proposed Clinical Grading System to Define Impaired Organ Function and Quality Of Life in Patients with von Hippel-Lindau (VHL) Disease in Japan - Abstract
Patients with von Hippel-Lindau (VHL) disease develop tumors and cysts in the central nervous system (CNS), retina, adrenal gland, kidney and pancreas. These tumors or cysts always require surgical treatment and cause different degrees of impairment in the affected organs. We developed a clinical grading system to evaluate impaired organ function and the quality of life (QOL) of VHL patients. Based on a previous grading system of intractable disease, impaired organ function is divided into five grades (grades 0, 1, 2, 3, and 4 for five affected organs) with QOL taken into consideration. The patient’s worst grade was regarded as their final clinical grade. The clinical grading of 46 patients was determined by a questionnaire. Our results showed that proportions of patients with grades 0, 1, 2, 3, 4 were 0% (0), 9% (2), 28% (15), 24% (12), 37% (17), respectively. If patients with two grade 3-affected organs are regarded as finally grade 4, then 46% of patients had grade 4. These results suggest that the organ function of approximately 50% of VHL patients is severely affected. Our study showed that our clinical grading system of VHL disease is relatively easy to use, and reflects the severity and QOL of VHL patients. The use of this system aids the provision of medical care and financial support from the Japanese health care system to VHL patients with severe impairment.