Kidney medicine · 2025
Natural History of Advanced Primary Hyperoxaluria Type 1: A Retrospective Study.
СтатьяНазвание и аннотация: Перевод в очереди
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Lieske John C, Groothoff Jaap W, Frishberg Yaacov, Garrelfs Sander F, Milliner Dawn S, Magen Daniella, Sellier-Leclerc Anne-Laure, Shasha-Lavsky Hadas, Bakkaloǧlu Sevcan, Lemoine Sandrine, Fuster Daniel G, Devresse Arnaud, Cytter-Kuint Ruth, Kajbaf Farshad, Du Weiming, Gansner John M
Аннотация
RATIONALE & OBJECTIVE: Characterize the natural history of advanced primary hyperoxaluria type 1 (PH1) in a multinational patient cohort.
STUDY DESIGN: A retrospective chart review.
SETTING & PARTICIPANTS: Patients, from participating medical centers in North America, Europe, and the Middle East, had ≥ 4 health care visits related to PH1 spanning ≥ 6 months (except deceased patients) on/after January 1, 2000, and ≥ 2 estimated glomerular filtration rate (eGFR) values ≤ 45 mL/min/1.73m2 (if age < 12 months, 2 serum creatinine values elevated for age) were included.
EXPOSURE: None (retrospective observational study).
OUTCOMES: Kidney function, liver and/or kidney transplantation, death, plasma oxalate, systemic oxalosis, emergent clinical events, and abnormal clinical laboratory values.
ANALYTICAL APPROACH: Patients were categorized as not on dialysis (Cohort A) and receiving hemodialysis (Cohort B). Patients could be in more than one cohort, but not during the same time.
RESULTS: Seventy patients were analyzed (up to 21 years of data; Cohort A, n = 54; Cohort B, n = 53). The median age at entry was 11.8 years (Cohort A) and 12.2 years (Cohort B). The eGFR slope was -2.8 mL/min/1.73m2/year (Cohort A). Patients underwent hemodialysis a median of 6 days/week (range, 3-7; Cohort B). Forty-two patients underwent liver and/or kidney transplantation (median age at first transplant, 15.3 years). Nineteen patients died (median age at death, 3.9 years [range, 2.2-34.9]), including 8 who received liver or liver-kidney transplants. Death occurred in 11 of 28 (39.3%) patients without transplant and 8 of 42 (19.0%) patients with transplant. Improvement in skeletal oxalosis after liver-kidney transplantation generally took > 1 year. Limited plasma oxalate and cardiac oxalosis data were available. The most common emergent clinical events were nephrolithiasis (Cohort A [nondialysis]) and fracture (Cohort B [hemodialysis]). The most commonly reported abnormal clinical laboratory values were bicarbonate, creatinine, and eGFR.
LIMITATIONS: Minimal follow-up in some patients; small sample for some endpoints.
CONCLUSIONS: Advanced PH1 is associated with high morbidity and mortality rates.
STUDY DESIGN: A retrospective chart review.
SETTING & PARTICIPANTS: Patients, from participating medical centers in North America, Europe, and the Middle East, had ≥ 4 health care visits related to PH1 spanning ≥ 6 months (except deceased patients) on/after January 1, 2000, and ≥ 2 estimated glomerular filtration rate (eGFR) values ≤ 45 mL/min/1.73m2 (if age < 12 months, 2 serum creatinine values elevated for age) were included.
EXPOSURE: None (retrospective observational study).
OUTCOMES: Kidney function, liver and/or kidney transplantation, death, plasma oxalate, systemic oxalosis, emergent clinical events, and abnormal clinical laboratory values.
ANALYTICAL APPROACH: Patients were categorized as not on dialysis (Cohort A) and receiving hemodialysis (Cohort B). Patients could be in more than one cohort, but not during the same time.
RESULTS: Seventy patients were analyzed (up to 21 years of data; Cohort A, n = 54; Cohort B, n = 53). The median age at entry was 11.8 years (Cohort A) and 12.2 years (Cohort B). The eGFR slope was -2.8 mL/min/1.73m2/year (Cohort A). Patients underwent hemodialysis a median of 6 days/week (range, 3-7; Cohort B). Forty-two patients underwent liver and/or kidney transplantation (median age at first transplant, 15.3 years). Nineteen patients died (median age at death, 3.9 years [range, 2.2-34.9]), including 8 who received liver or liver-kidney transplants. Death occurred in 11 of 28 (39.3%) patients without transplant and 8 of 42 (19.0%) patients with transplant. Improvement in skeletal oxalosis after liver-kidney transplantation generally took > 1 year. Limited plasma oxalate and cardiac oxalosis data were available. The most common emergent clinical events were nephrolithiasis (Cohort A [nondialysis]) and fracture (Cohort B [hemodialysis]). The most commonly reported abnormal clinical laboratory values were bicarbonate, creatinine, and eGFR.
LIMITATIONS: Minimal follow-up in some patients; small sample for some endpoints.
CONCLUSIONS: Advanced PH1 is associated with high morbidity and mortality rates.