Administration options for pegfilgrastim prophylaxis: patient and physician preferences from a cross-sectional survey.

OBJECTIVE: Although clinical guidelines recommend administration of pegfilgrastim 1-4 days after a myelosuppressive chemotherapy cycle to decrease the incidence of febrile neutropenia (FN), some physicians administer pegfilgrastim on the same day as chemotherapy administration. A novel on-body injector (OBI) that automatically delivers pegfilgrastim the day after chemotherapy is also available. Our objective was to estimate patient and physician preferences among the pegfilgrastim administration options. METHODS: We conducted a cross-sectional survey of patients receiving pegfilgrastim and physicians prescribing pegfilgrastim. Respondents' preferences for pegfilgrastim administration options were elicited using direct elicitation; the relative importance of features associated with the options was estimated in a point-allocation exercise. Physicians considered two hypothetical patient profiles when completing the exercises. RESULTS: The samples included 200 patients and 200 physicians. Patients generally preferred the administration option with which they had experience. Among patients, 48.5% with previous in-clinic injections 24 hours after chemotherapy preferred this option; 56.8% with previous OBI administration preferred this option. For a clinically compromised patient, 37.5% of physicians preferred an in-clinic injection option; 49.5% preferred the OBI. For a less compromised patient, 55.5% preferred an in-clinic injection option; 28.0% preferred the OBI. Avoiding the need to return to the clinic was chosen most often as the most important treatment feature for patients and physicians. CONCLUSIONS: Patients and physicians identified that returning clinic visits for pegfilgrastim administration may be burdensome. A potential solution to mitigate this burden is the OBI, which allows adherence to the labeled use of pegfilgrastim without return visits to the clinic.

Patients' and caregivers' maximum acceptable risk of death for non-curative gene therapy to treat Duchenne muscular dystrophy.

BACKGROUND: Gene therapy offers an etiologically targeted treatment for genetic disorders. Little is known about the acceptance of mortality risk among patients with progressive, fatal conditions. We assessed patients' and caregivers' maximum acceptable risk (MAR) of mortality for gene therapy when used to treat Duchenne muscular dystrophy (DMD). METHODS: The threshold technique was used to assess tolerance for mortality risks using a hypothetical vignette. Gene therapy was described as non-curative and resulting in a slowing of progression and with a 10-year benefit duration. MAR was analyzed using interval regression for gene therapy initiated "now"; in the last year of walking well; in the last year of being able to bring arms to mouth; and in newborns (for caregivers only). RESULTS: Two hundred eighty-five caregivers and 35 patients reported the greatest MAR for gene therapy initiated in last year of being able to lift arms (mean MAR 6.3%), followed by last year of walking well (mean MAR 4.4%), when used "now" (mean MAR 3.5%), and when used in the newborn period (mean MAR 2.1%, caregivers only). About 35% would accept ≥200/2000 risk in the last year of being able to lift arms. Non-ambulatory status predicted accepting 1.8 additional points in MAR "now" compared with ambulatory status (p = 0.010). Respondent type (caregiver or patient) did not predict MAR. CONCLUSION: In this first quantitative study to assess MAR associated with first-generation DMD gene therapy, we find relatively high tolerance for mortality risk in response to a non-curative treatment scenario. Risk tolerance increased with disease progression. Patients and caregivers did not have significantly different MAR. These results have implications for protocol development and shared decision making.