Patient engagement and study design of PROP UP: A multi-site patient-centered prospective observational study of patients undergoing hepatitis C treatment

Contemp Clin Trials. 2017 Jun;57:58-68. doi: 10.1016/j.cct.2017.03.013. Epub 2017 Mar 22.

Abstract

Background: New highly efficacious direct-acting antiviral (DAA) therapies are available to treat chronic hepatitis C viral (HCV) infection. Real-world, patient-centered data on harms and benefits associated with these therapies are needed.

Methods: PROP UP is a multi-center prospective observational study that plans to enroll 1600 patients starting treatment with recently-approved DAA regimens. Informed by extensive input from a HCV patient engagement group who prioritized outcomes most important to them, patient-reported outcomes will be characterized using surveys at five time points: Baseline (T1), treatment week 4 (T2), end of treatment (T3), 12weeks post-treatment (T4), 12months post-treatment (T5).

Outcomes: (1) Changes in side effects, functioning, pre-existing conditions, and out-of-pocket costs during therapy (T1 vs T2/T3); (2) Medication adherence in relation to a history of mental health/substance abuse, treatment regimens, pill burden, reasons for missed doses, and cure rates; (3) Short term impact of cure on functioning and amelioration of symptoms (T1 vs T4); (4) Long-term treatment harms or benefits of cure on symptoms, side effects, pre-existing conditions, and functioning (T1 vs T5). Similarities between regimens will be examined where comparisons are appropriate and meaningful.

Conclusion: PROP UP complements previous clinical trials by focusing on patient-reported outcomes in a representative sample of patients treated in clinical practice, by collaborating with a patient engagement group, by characterizing the experiences of vulnerable subgroups, and by investigating long-term harms and benefits of treatments. PROP UP is designed to provide novel and detailed information to support informed decision-making for patients and providers contemplating HCV treatment (PCORI CER-1408-20,660; NCT02601820).

Keywords: Direct acting antiviral (DAA); Hepatitis; Liver; Patient-centered outcomes research (PCOR); Patient-reported outcomes.

Publication types

  • Multicenter Study
  • Observational Study

MeSH terms

  • Adult
  • Antiviral Agents / adverse effects
  • Antiviral Agents / therapeutic use*
  • Benzimidazoles / adverse effects
  • Benzimidazoles / therapeutic use
  • Benzofurans / adverse effects
  • Benzofurans / therapeutic use
  • Carbamates / adverse effects
  • Carbamates / therapeutic use
  • Drug Combinations
  • Female
  • Fluorenes / adverse effects
  • Fluorenes / therapeutic use
  • Hepatitis C, Chronic / drug therapy*
  • Heterocyclic Compounds, 4 or More Rings / adverse effects
  • Heterocyclic Compounds, 4 or More Rings / therapeutic use
  • Humans
  • Imidazoles / adverse effects
  • Imidazoles / therapeutic use
  • Macrocyclic Compounds / adverse effects
  • Macrocyclic Compounds / therapeutic use
  • Male
  • Patient Participation*
  • Patient-Centered Care / methods
  • Prospective Studies
  • Quinoxalines / adverse effects
  • Quinoxalines / therapeutic use
  • Ritonavir / adverse effects
  • Ritonavir / therapeutic use
  • Sofosbuvir / adverse effects
  • Sofosbuvir / therapeutic use
  • Sulfonamides / adverse effects
  • Sulfonamides / therapeutic use
  • Treatment Outcome
  • Uracil / adverse effects
  • Uracil / analogs & derivatives
  • Uracil / therapeutic use
  • Uridine Monophosphate / adverse effects
  • Uridine Monophosphate / analogs & derivatives
  • Uridine Monophosphate / therapeutic use

Substances

  • Antiviral Agents
  • Benzimidazoles
  • Benzofurans
  • Carbamates
  • Drug Combinations
  • Fluorenes
  • Heterocyclic Compounds, 4 or More Rings
  • Imidazoles
  • Macrocyclic Compounds
  • Quinoxalines
  • Sulfonamides
  • Viekira Pak
  • elbasvir-grazoprevir drug combination
  • ledipasvir, sofosbuvir drug combination
  • sofosbuvir-velpatasvir drug combination
  • Uracil
  • Uridine Monophosphate
  • daclatasvir
  • Ritonavir
  • Sofosbuvir

Associated data

  • ClinicalTrials.gov/NCT02601820