Current management and use of oral immunotherapy in the United States for patients with peanut allergy.

Background: Peanut allergy is a major health burden in the United States. Treatment is limited to avoidance and acute reaction management. No drug or medical product is approved for use as a peanut oral immunotherapy (POIT) agent. Objective: To examine peanut allergy diagnosis and treatment, peanut challenge protocols, nonpublished POIT approaches, POIT practice requirements and logistical considerations, and barriers to providing POIT. Methods: Qualitative in-depth telephonic interviews were conducted with 34 allergists and nurse food allergy specialists across the United States between April and June 2016. Interviewed clinicians managed > 100 patients with peanut allergy per year; 50% of the interviewed allergists offered POIT in clinical studies or used self-developed approaches. Results: The physicians consistently reported conducting food challenges in 5-10% of patients to confirm a peanut allergy diagnosis. The allergists who offered POIT described using a variety of approaches. Areas of divergence included patient selection (ages, 4-7 years), peanut material (crushed peanuts, peanut flour, peanut protein, peanut butter, peanut extract), starting and ending doses, and updosing intervals (1 to 2 weeks). Generally, POIT administration and observation occupied an examination room for up to 2 hours; some practices reported accommodating 2 to 5 patients who received POIT simultaneously. Among physicians who did not offer POIT, barriers included medicolegal risks and the lack of a U.S. Food and Drug Administration (FDA) approved therapy. Conclusion: Although POIT is currently not supported in treatment guidelines, some allergists have developed experimental POIT approaches to support patient needs. In the absence of a product that has approval by the FDA, European Medicines Agency (EMA) or other national competent authority, substantial variability in POIT approaches exists. Although logistical factors are not major obstacles to adoption, POIT dose preparation can be perceived as burdensome, and observation requires a dedicated staff. All the physicians interviewed suggested a need for effective, FDA-approved, disease-modifying treatments.

Sorafenib use for recurrent hepatocellular cancer after resection or transplantation: Observations from a US regional analysis of the GIDEON registry.

Treatment of unresectable recurrent hepatocellular carcinoma (HCC) in patients who recur after resection or orthotopic liver transplantation (OLT) remains a clinical challenge. One option is sorafenib, although little is known about its safety and tolerance in this unique patient population; therefore, we analyzed patients who underwent prior surgical resection and/or OLT and were treated with sorafenib in US cohort of GIDEON registry. In US, 645 patients were enrolled; 553 for intent to treat and 563 for safety. Data were analyzed in the safety population of 479 patients no surgery and 56 for resection or OLT. Forty-one patients underwent resection prior to the initiation of sorafenib, 15 patients had previously received an OLT, and 6 patients had both resection and OLT. Initial low starting doses (400 mg/day) were observed for more patients with prior OLT (71%) than prior resection (36%), resection and OLT (50%), concomitant OLT (25%), and no surgery (36%). Most AEs occurred in the first 4 weeks of treatment. Drug-related AEs were higher in patients with prior resection (87%), prior OLT (100%), or both (100%) than in patients with concomitant OLT (63%) or no surgery (70%). However, incidence of AEs resulting in permanent discontinuation were similar in all groups (19-38%).

Use of Transarterial Chemoembolization (TACE) and Sorafenib in Patients with Unresectable Hepatocellular Carcinoma: US Regional Analysis of the GIDEON Registry.

BACKGROUND: Global Investigation of Therapeutic Decisions in Hepatocellular Carcinoma and of Treatment with Sorafenib (GIDEON) is a worldwide, prospective, non-interventional study to evaluate the safety of sorafenib in a variety of patient subsets. METHODS: Eligible patients had unresectable hepatocellular carcinoma for whom the decision had been made to treat with sorafenib. Treatment strategies were instituted at the physician's discretion. Patient and disease characteristics, treatment practices, incidences of adverse events (AEs), and overall survival were collected. RESULTS: In the United States, 563 patients were evaluable for safety. Subgroup analysis was performed for patients who underwent transarterial chemoembolization (TACE) prior to the initiation of sorafenib (group A, n=158), after the initiation of sorafenib only (group B, n=29), both (group C, n=38), or did not undergo TACE (n=318). Patient demographics were similar across the groups. In group A, 29% had Child-Pugh score B or C at diagnosis, and 19% had Barcelona Clinic Liver Cancer tumor stage C or D. In group B, 48% had Child-Pugh score B or C at study entry, and 31% had BCLC stage C or D. The majority of patients in all groups initially received full-dose sorafenib. Incidences of grade ≥3 drug-related AEs were 30%, 17%, and 16% in groups A, B, and C, respectively, and 22% in patients who did not undergo TACE. No new safety signals emerged. CONCLUSIONS: The results from GIDEON reaffirm that sorafenib can be safely used in the context of TACE.