Kidney transplantation improves the clinical outcomes of Acute Intermittent Porphyria

Mol Genet Metab. 2020 Sep-Oct;131(1-2):259-266. doi: 10.1016/j.ymgme.2020.08.004. Epub 2020 Sep 2.

Abstract

Background: Acute Intermittent Porphyria (AIP) is a rare inherited autosomal dominant disorder of heme biosynthesis. Porphyria-associated kidney disease occurs in more than 50% of the patients with AIP, and end stage renal disease (ESRD) can be a devastating complication for AIP patients. The outcomes of AIP patients after kidney transplantation are poorly known.

Methods: We examined the outcomes of 11 individuals with AIP, identified as kidney transplant recipients in the French Porphyria Center Registry.

Results: AIP had been diagnosed on average 19 years before the diagnosis of ESRD except for one patient in whom the diagnosis of AIP had been made 5 years after the initiation of dialysis. Median follow-up after transplantation was 9 years. A patient died 2 months after transplantation from a cardiac arrest and a patient who received a donation after cardiac death experienced a primary non-function. No rejection episode and no noticeable adverse event occurred after transplantation. Serum creatinine was on average 117 μmol/l, and proteinuria <0.5 g/l in all patients at last follow up. All usually prescribed drugs after transplantation are authorized except for trimethoprim/sulfamethoxazole. Critically, acute porphyria attacks almost disappeared after kidney transplantation, and skin lesions resolved in all patients.

Conclusion: Kidney transplantation is the treatment of choice for AIP patients with ESRD and dramatically reduces the disease activity.

Keywords: Acute Intermittent Porphyria; Aminolevulinic acid; Chronic kidney disease; End-stage renal disease; Kidney transplantation; Porphobilinogen; Porphyria cutanea tarda; Porphyrin precursors.

MeSH terms

  • Adult
  • Female
  • Heme / biosynthesis
  • Heme / genetics
  • Humans
  • Kidney / pathology*
  • Kidney Failure, Chronic / complications
  • Kidney Failure, Chronic / genetics
  • Kidney Failure, Chronic / pathology
  • Kidney Failure, Chronic / therapy*
  • Kidney Transplantation*
  • Male
  • Middle Aged
  • Porphyria, Acute Intermittent / complications
  • Porphyria, Acute Intermittent / genetics
  • Porphyria, Acute Intermittent / pathology
  • Porphyria, Acute Intermittent / therapy*
  • Treatment Outcome
  • Young Adult

Substances

  • Heme