RESUMEN
The use of iron supplementation for anemia in erythropoietic protoporphyria (EPP) is controversial with both benefit and deterioration reported in single case reports. There is no systematic study to evaluate the benefits or risks of iron supplementation in these patients. We assessed the potential efficacy of oral iron therapy in decreasing erythrocyte protoporphyrin (ePPIX) levels in patients with EPP or X-linked protoporphyria (XLP) and low ferritin in an open-label, single-arm, interventional study. Sixteen patients (≥18 years) with EPP or XLP confirmed by biochemical and/or genetic testing, and serum ferritin ≤30 ng/mL were enrolled. Baseline testing included iron studies, normal hepatic function, and elevated plasma porphyrins and ePPIX levels. Oral ferrous sulfate 325 mg twice daily was administered for 12 months. The primary efficacy outcome was the relative difference in total ePPIX level between baseline and 12 months after starting treatment with iron. Secondary measures included improvement in serum ferritin, plasma porphyrins, and clinical symptoms. Thirteen patients had EPP (8 females, 5 males) and 3 had XLP (all females) and the mean age of participants was 38.8 years (SD 14.5). Ten patients completed all study visits limiting interpretation of results. In EPP patients, a transient increase in ePPIX levels was observed at 3 months in 9 of 12 (75%) patients. Iron was discontinued in 2 of these patients after meeting the protocol stopping rule of a 35% increase in ePPIX. Seven patients withdrew before study end. Ferritin levels increased on iron replacement indicating an improvement in iron status. A decrease in ePPIX was seen in both XLP patients who completed the study (relative difference of 0.67 and 0.5 respectively). No substantial changes in ePPIX were seen in EPP patients at the end of the study (n = 8; median relative difference: -0.21 (IQR: -0.44, 0.05). The most common side effects of iron treatment were gastrointestinal symptoms. Hepatic function remained normal throughout the study. Our study showed that oral iron therapy repletes iron stores and transiently increases ePPIX in some EPP patients, perhaps due to a transient increase in erythropoiesis, and may decrease ePPIX in XLP patients. Further studies are needed to better define the role of iron repletion in EPP. Trial registration: NCT02979249.
RESUMEN
BACKGROUND: As tumor burden increases in colorectal cancer, treatment complexity progresses from colectomy to hepatectomy and lastly to cytoreductive surgery with heated intraperitoneal chemotherapy (CRS-HIPEC). The aim of this study was to evaluate whether disparities exist in the access to progressively more complex surgical treatment options. METHODS: Patients undergoing surgery for colorectal cancer were grouped by treatment type: group 1 (n = 224) underwent colectomy for nonmetastatic disease, group 2 (n = 112) underwent hepatectomy for liver metastasis, and group 3 (n = 112) underwent CRS-HIPEC for carcinomatosis. RESULTS: Whites were predominant in the HIPEC group (71.4 %) compared to the hepatectomy (67.9 %) and colectomy (57.6 %) groups (p = 0.025). The majority of the privately insured patients were in the HIPEC group (70.5 %) compared to the hepatectomy (56.2 %) and colectomy (30.4 %) groups (p < 0.0001). Distance traveled to the hospital was farthest on average in the HIPEC group (104.6 ± 258.3 km) compared to the hepatectomy (29.0 ± 28.0 km) or colectomy (26.4 ± 66.2 km) group (p < 0.0001). Mean household income also varied between the three groups, with HIPEC patients earning $56,957 (±24,124), hepatectomy patients earning $56,999 (±28,588), and colectomy patients earning ($51,518 ± 24,201) (p = 0.0503) on average per year. The HIPEC cohort contained a higher proportion of English speakers (90.2 %) than the other groups (hepatectomy 87.9 %, colectomy 85.3 %); however, this difference was not statistically significant (p = 0.43). CONCLUSIONS: CRS-HIPEC is not accessed equally across all socioeconomic groups. Patients undergoing HIPEC were most often white, English speaking, and privately insured; had a higher mean income; and had traveled the greatest distances on average to access surgical care.