Clinical features and outcomes of 98 children and adults with dense deposit disease
BACKGROUND: Dense deposit disease (DDD) is an ultra-rare renal disease. METHODS: In the study reported here, 98 patients and their families participated in a descriptive patient-centered survey using an online research format. Reports were completed by patients (38%) or their parents (62%). Age at diagnosis ranged from 1.9 to 38.9 years (mean 14 years). RESULTS: The majority of patients presented with proteinuria and hematuria; 50% had hypertension and edema. Steroids were commonly prescribed, although their use was not evidence-based. One-half of the patients with DDD for 10 years progressed to end-stage renal disease (ESRD), with young females having the greatest risk for renal failure. Of first allografts, 45% failed within 5 years, most frequently due to recurrent disease (70%). Type 1 diabetes (T1D) was present in over 16% of families, which represents a 116-fold increase in incidence compared with the general population (p < 0.001). CONCLUSIONS: Based on these findings, we suggest that initiatives are needed to explore the high incidence of T1D in family members of DDD patients and the greater risk for progression to ESRD in young females with DDD. These efforts must be supported by sufficient numbers of patients to establish evidence-based practice guidelines for disease management. An international collaborative research survey should be implemented to encourage broad access and participation.
Der-Fa Lu, M. Moon, L. D. Lanning, Ann Marie McCarthy, and R. J. Smith. "Clinical features and outcomes of 98 children and adults with dense deposit disease" Pediatric Nephrology (Berlin, Germany) 27.5 (2012): 773-781.
Available at: http://works.bepress.com/ann_mccarthy/90
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