High incidence of progression to chronic renal insufficiency in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Peter Hillmen; Modupe Elebute; Richard J. Kelly; Álvaro Urbano-Ispizúa; Russell P. Rother; Chieh‐Lin Fu; Paul Browne

doi:10.7490/f1000research.1092126.1

July 27, 2012F1000ResearchOpen Access

High incidence of progression to chronic renal insufficiency in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

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Key Points

To assess the incidence, underdiagnosis, and risk factors of chronic renal insufficiency in patients with paroxysmal nocturnal hemoglobinuria, and evaluate eculizumab safety and renal outcomes.
Screened 195 hemolytic paroxysmal nocturnal hemoglobinuria patients enrolled across multinational clinical trials (Phase 2, TRIUMPH, and SHEPHERD).
Evaluated glomerular filtration rate (GFR), previous diagnoses of acute renal failure (ARF) and chronic renal insufficiency (CRI), disease history, and response to eculizumab treatment.
CRI was identified in 21% (40/195) of patients at screening, including 10 with severe CRI (GFR ≤30 ml/min), yet only 25% (10/40) had a prior clinical diagnosis of CRI.
Patients with CRI had longer median disease duration (8.8 vs 5.1 years) and were significantly more likely to have disease for ≥10 years (48% vs 28%, P=0.02) and thrombosis history ≥10 years (13% vs 3%, P=0.03).
Eculizumab was well-tolerated, reduced median LDH from 1783 to 331 U/L, resolved CRI classification in 10% of affected patients, and reduced recurrent ARF from 14 pre-treatment patients to 1 during treatment.

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Implication

Multinational clinical trial analysis demonstrates high rates of chronic renal insufficiency in paroxysmal nocturnal hemoglobinuria, highlighting frequent underdiagnosis.

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Cite This Study

Hillmen et al. (2012) studied this question.

synapsesocial.com/papers/6a94cdb60664514f7d5e227d https://doi.org/10.7490/f1000research.1092126.1

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High incidence of progression to chronic renal insufficiency in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Key Points

No abstract yet

Implication

Discussion

Cite This Study

High incidence of progression to chronic renal insufficiency in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Key Points

No abstract yet

Implication

Discussion

Cite This Study

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