Is liver transplantation for intractable attacks of porphyria a viable treatment in a developing country?
Authors: Daniel A. Jaramillo‐Calle, Omar Castaño Quintero, Juan‐Ignacio Marín, Alicia M. Cock‐Rada, Oscar Santos Sánchez, Octavio Muñoz, Juan‐Carlos Restrepo
Journal: Hepatology (2017)

Potential conflict of interest: Nothing to report. To the editor: We read with great interest the guideline for the long‐term management of acute hepatic porphyrias by Balwani et al.1 The authors mentioned that orthotopic liver transplantation (OLT) has been used for intractable attacks that are refractory to heme therapy. The first successful OLT for this indication was reported in 2004,2 and then other cases were reported from European and American developed countries. However, there is no published evidence that this procedure has been successfully performed in a developing country. Therefore, we want to contribute by reporting the first experience in Latin America. A 30‐year‐old woman with asthma, allergic rhinitis, type 2 diabetes, and endometriosis was diagnosed with acute intermittent porphyria at age 27. Over 3 years, she was hospitalized 14.5 times per year on average, with a mean hospital stay of 11 days, due to recurrent and severe attacks which were mostly precipitated by the menstrual cycle. Gonadorelin analogues (Zoladex, AstraZeneca) and monthly prophylactic infusions of heme arginate (Normosang, Orphan Europe) were administered without bringing about any improvement. The patient received 193 heme doses (48.250 mg heme/4,381.1 mg iron) and developed secondary iron overload (ferritin 1,090 ng/mL), multiple venous thrombosis with loss of vascular access, catheter‐related bloodstream infections, and anaphylaxis. For the serious allergic reaction, she was pretreated with systemic corticosteroids during subsequent attacks to receive heme, which caused an exogenous Cushing syndrome. She was placed on the waiting list for an OLT as an exception and waited for 1 year (12 times the usual waiting average) because patients in worse conditions were always prioritized. As complications, she had postextubation respiratory insufficiency caused by anesthetic drug recirculation, early acute cell rejection, and cytomegalovirus syndrome, all of which were successfully managed. Postoperative anticoagulation was administered based on reports of a high rate of hepatic arterial thrombosis at acute hepatic porphyria receptors.3 Urinary levels of porphobilinogen and 5‐aminolevulinic acid normalized within 48 hours post‐OLT, and there have been no symptoms after 6 months. OLT is a viable treatment for intractable attacks in developing countries. One of the main expectable difficulties in these countries is very prolonged waiting times because most patients have structurally healthy livers and regular prioritization scores are not applicable. Hence, attempting to identify patients at high risk of becoming refractory to heme therapy is essential to avoid permanent neurological sequelae or death during the waiting period. Patients with multiple comorbidities might be more vulnerable to recurrent attacks, so it is essential to closely monitor and control their illnesses. This case supports the curative role of OLT in acute hepatic porphyrias.