Ponatinib was associated with significantly longer overall survival than allogeneic stem cell transplantation (alloSCT) in patients with chronic myeloid leukemia in chronic phase (CP-CML) that harbor the T315I mutation.1

AlloSCT is considered standard treatment for patients with accelerated or blast phase CML, de novo Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL), or patients with CP-CML who are resistant or intolerant to at least 2 tyrosine kinase inhibitors (TKIs). Ponatinib is indicated for the treatment of patients with CML or Ph+ ALL who have a positive BCR-ABL1 T315I mutation or for when there is no other suitable TKI available. Therefore, ponatinib currently represents a suitable alternative to alloSCT in patients harboring a T315I mutation, but outcomes between patients treated with these 2 approaches have not been compared.

For the study, researchers pooled data from the phase 2 PACE trial and the European Bone Marrow Transplant registry to indirectly compare the 2 treatment strategies. A total of 128 patients who received ponatinib and 56 who underwent alloSCT were included.

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Results showed that among patients with CP-CML, median overall survival had not been reached in the ponatinib group and was 103.3 months in the alloSCT group (HR, 0.37; 95% CI, 0.16 – 0.84; P = .017). In contrast, there was no significant difference in median overall survival between the 2 treatment groups in patients with CML in accelerated phase (HR, 0.90; 95% CI, 0.20 – 4.10; P = .889) and was significantly shorter in those in blast phase who had received ponatinib vs alloSCT (HR, 2.29; 95% CI, 1.08 – 4.82; P = .030).

In regard to patients with Ph+ ALL, those treated with ponatinib had nominally shorter median overall survival compared with those who had received alloSCT (HR, 2.77; 95% CI, 0.73 – 10.56; P = .136).

Although follow-up remains short to date, ponatinib could represent a promising therapeutic alternative in this setting.


  1. Nicolini FE, Basak GW, Kim D-W, et al. The impact of ponatinib versus allogeneic stem cell transplant (SCT) on outcomes in patients with chronic myeloid leukemia (CML) or Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) with the T315I mutation [abstract]. https://ash.confex.com/ash/2015/webprogram/Paper84244.html. Updated December 7, 2015. Accessed January 11, 2016.