Phase I trial of pegylated interferon-α-2b in young patients with plexiform neurofibromas
RI Jakacki, E Dombi, DM Potter, S Goldman, JC Allen… - Neurology, 2011 - AAN Enterprises
Objective: Interferon has antiproliferative and antiangiogenic properties. We sought to
evaluate preliminary efficacy and determine the recommended phase II dose (RP2D) for
pegylated interferon-α-2b (PI) in patients with unresectable progressive or symptomatic
plexiform neurofibromas (PN). Methods: PI was administered weekly in cohorts of 3–6
patients during the dose-finding phase and continued for up to 2 years. Twelve patients
were treated at the RP2D to further evaluate toxicity and activity. Results: Thirty patients …
evaluate preliminary efficacy and determine the recommended phase II dose (RP2D) for
pegylated interferon-α-2b (PI) in patients with unresectable progressive or symptomatic
plexiform neurofibromas (PN). Methods: PI was administered weekly in cohorts of 3–6
patients during the dose-finding phase and continued for up to 2 years. Twelve patients
were treated at the RP2D to further evaluate toxicity and activity. Results: Thirty patients …
Phase II trial of pegylated interferon alfa-2b in young patients with neurofibromatosis type 1 and unresectable plexiform neurofibromas
RI Jakacki, E Dombi, SM Steinberg, S Goldman… - Neuro …, 2017 - academic.oup.com
Background. There is no proven medical therapy for plexiform neurofibromas (PNs). We
undertook a phase II trial of pegylated interferon (PI) to evaluate response and time to
progression (TTP). Methods. PI was administered as a subcutaneous injection to patients
with neurofibromatosis type 1‒related PN, stratified by the presence of symptoms
(asymptomatic: stratum 1, symptomatic: stratum 2) or documented imaging progression
(stratum 3). Patients in strata 1 and 2 received PI for up to one year if stable, 2 years for …
undertook a phase II trial of pegylated interferon (PI) to evaluate response and time to
progression (TTP). Methods. PI was administered as a subcutaneous injection to patients
with neurofibromatosis type 1‒related PN, stratified by the presence of symptoms
(asymptomatic: stratum 1, symptomatic: stratum 2) or documented imaging progression
(stratum 3). Patients in strata 1 and 2 received PI for up to one year if stable, 2 years for …