PEGylated interferon alfa-2b (Anhui Anke Biotechnology)

We evaluated the tolerability and efficacy of pegylated interferon alfa‐2B (peg‐IFNα; PegIntron, MSD) combined with nilotinib in the Australasian Leukaemia and Lymphoma Group CML11 (Pinnacle) study. This phase II study started patients on nilotinib 300 mg twice daily. Subcutaneous peg‐IFNα was added at 30–50 50 μg/week from 3 months until 24 months as tolerated. Sixty patients were enrolled with a median age of 48.5 years (range 19–72); 45% were female. With a median follow‐up of 60 months, 40 patients (67%) remain on study. The proportion of patients who received ≥50% and ≥85% of their assigned peg‐IFNα doses were 58% and 35% respectively. Common reasons for peg‐IFNα discontinuation were mood disturbance (5), thyroid disease (4) and myalgia (4). The cumulative incidence of Major Molecular Response (MMR, BCR::ABL1 ≤0.1%) was 87% by 12 months; Molecular Response 4.5 (MR4.5, BCR::ABL1 ≤0.0032%) incidence at 24 and 60 months was 55% and 82% respectively. Thirty‐seven patients (62%) had MR4.5 for >24 months, 14 of whom attempted treatment‐free remission (TFR); 13 remained in TFR at a median follow‐up of 32 months. CML11 demonstrated that peg‐IFNα with nilotinib leads to high rates of molecular response, with tolerability similar to prior studies. Trial registration ANZCTRN12612000851864.