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Protoporphyria is a subtype of porphyria characterized primarily by painful phototoxic skin reactions after light exposure at specific wavelengths. Historically, phototherapy is not contraindicated in patients with protoporphyria since there have not been any reports of phototoxic reactions. However, patients with protoporphyria are advised to avoid direct sunlight. In this case report, we describe a neonate not known to have X-linked protoporphyria who received phototherapy for 1 to 2 hours. Within hours after initiation of phototherapy, this neonate developed a life-threatening reaction consisting of rash over the distribution of phototherapy, acute liver failure with coagulopathy, diffuse hypotonia with diaphragmatic failure, and subsequent acute respiratory failure that required mechanical ventilation. As in this case, patients with protoporphyria-related acute liver failure can have signs and symptoms similar to that of an acute hepatic porphyria attack. Neither neonatal reactions to phototherapy nor liver failure temporally associated with phototherapy have been reported in patients with X-linked protoporphyria. Early recognition of this entity is crucial in light of potential life-threatening complications. Therefore, providers must react quickly when neonates have abnormal reactions to phototherapy and consider protoporphyria in the differential diagnosis.
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http://dx.doi.org/10.1542/peds.2023-064724 | DOI Listing |
JAAD Int
October 2025
Department of Medicine, Massachusetts General Hospital, Boston, Massachusetts.
J Dermatol
August 2025
Disc Medicine, Watertown, Massachusetts, USA.
Erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) are rare metabolic disorders causing phototoxicity, potential liver damage, and significant burden in all facets of life. We sought to quantify symptoms, health-related quality of life (HRQoL), work/school productivity, and healthcare utilization (HCU) of individuals with protoporphyria using the EPP Life Impact and Genetic Health Trajectory (LIGHT) study. A cross-sectional, online questionnaire administered to 164 adults and 33 adolescents with protoporphyria aged ≥ 12 years, included validated patient-reported outcome measures and original items assessing symptoms, HRQoL, productivity, and HCU.
View Article and Find Full Text PDFJ Clin Invest
July 2025
Department of Pathology, Boston Children's Hospital and Harvard Medical School, Boston, United States of America.
Erythropoietic protoporphyria (EPP) is a genetic disorder typically resulting from decreased ferrochelatase (FECH) activity, the last enzyme in heme biosynthesis. Patients with X-linked protoporphyria (XLPP) have an overlapping phenotype caused by increased activity of 5-aminolevulinic acid synthase 2 (ALAS2), the first enzyme in erythroid heme synthesis. In both cases, protoporphyrin IX (PPIX) accumulates in erythrocytes and secondarily in plasma and tissues.
View Article and Find Full Text PDFErythropoietic protoporphyria (EPP) and X-linked protoporphyria are metabolic disorders that cause skin phototoxicity and potential liver damage. We compared symptoms and impacts of phototoxic reactions, health-related quality of life, and healthcare utilization (HCU) between individuals with and without self-reported liver disease (elevated liver enzymes or liver fibrosis) using an online questionnaire containing validated patient-reported outcome measures and original items. Among 164 participants, 15.
View Article and Find Full Text PDFHepatol Commun
March 2025
Department of Population Health Science and Policy, Center for Biostatistics, Icahn School of Medicine at Mount Sinai, New York City, New York, USA.
Background: Erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) are characterized by the accumulation of protoporphyrin in the marrow, erythrocytes, plasma, skin, and liver, and present clinically with painful cutaneous phototoxicity. Liver abnormalities have been reported in over 25% of patients with EPP. Further characterization of liver involvement in protoporphyria is needed.
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