Bridge to Transplant: Ponatinib and Leukemia Treatment

Hope for Relapsed Leukemia: Can Ponatinib Bridge the Gap to Successful Transplants?

Elliot Brynn in Health & Wellness December 2025 • 4 min read.

"A new study explores how ponatinib, a targeted therapy, can help patients with Philadelphia chromosome-positive leukemia reach potentially life-saving bone marrow transplants."

Outcomes for patients battling relapsed or refractory Philadelphia chromosome-positive (Ph+) leukemia have historically been bleak. However, the emergence of new treatments, including ponatinib, offers renewed hope. This third-generation tyrosine kinase inhibitor (TKI) demonstrates a potent effect against various mutations, including the challenging T315I mutation.

While ponatinib has shown promise in treating resistant chronic phase chronic myeloid leukemia (CP-CML), its effectiveness in advanced Ph+ leukemia requires innovative strategies. One such approach involves combining ponatinib with other therapies to facilitate a bridge to allogeneic hematopoietic cell transplantation (HCT), also known as a bone marrow transplant.

A recent single-center retrospective study sheds light on this approach, investigating the use of ponatinib as a salvage therapy to enable HCT in patients with relapsed or refractory Ph+ leukemia. The study highlights the potential of ponatinib to control the disease and create a path toward successful transplantation.

Ponatinib to the Rescue: Study Design and Patient Population

Bridge to Transplant: Ponatinib and Leukemia Treatment

The study, conducted at a single center, retrospectively analyzed consecutive patients who received ponatinib as salvage therapy to facilitate allogeneic hematopoietic cell transplantation (HCT) for relapsed or refractory Ph-leukemia between January 2017 and July 2018.

A total of ten patients were included in the study, comprising seven with Ph+ acute lymphoblastic leukemia (ALL) and three with chronic phase (CP)/accelerated phase chronic myeloid leukemia (CML). Notably, eight patients had previously been treated with a single tyrosine kinase inhibitor (TKI) before receiving ponatinib.

Mutation Detection: Mutation analysis revealed that eight patients had mutations in the tyrosine kinase domain, with seven exhibiting the T315I mutation.
Ponatinib Dosage: The median dose of ponatinib administered was 15 mg, with a median treatment duration of 7 weeks (ranging from 4 to 23 weeks).
Bridging to HCT: The median time from the start of ponatinib treatment to HCT was 54 days (ranging from 35 to 175 days).

The study's findings suggest that salvage therapy with low-dose ponatinib is safe and effective for patients with relapsed or refractory Ph-leukemia, potentially enabling a bridge to HCT.

A Promising Path Forward

This study offers a beacon of hope for patients with relapsed or refractory Ph+ leukemia. Salvage therapy with low-dose ponatinib appears to be a safe and effective strategy for controlling the disease and facilitating a bridge to potentially life-saving HCT. While further investigations are needed, these findings suggest that ponatinib can play a crucial role in improving outcomes for this challenging patient population. With continued research and refinement of treatment strategies, the future looks brighter for individuals battling Ph+ leukemia.

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This article is based on research published under:

DOI-LINK: 10.1007/s12185-018-02571-0, Alternate LINK

Title: Salvage And Bridging To Allogeneic Hematopoietic Cell Transplantation With Ponatinib In Patients With Relapsed Or Refractory Philadelphia Chromosome-Positive Leukemia

Subject: Hematology

Journal: International Journal of Hematology

Publisher: Springer Science and Business Media LLC

Authors: Takayoshi Tachibana, Satoshi Koyama, Taiki Andou, Yasufumi Ishiyama, Masatsugu Tanaka, Hideaki Nakajima, Heiwa Kanamori

Published: 2018-12-03

Everything You Need To Know

What is ponatinib and why is it important in treating leukemia?

Ponatinib is a third-generation tyrosine kinase inhibitor (TKI) that has a potent effect against various mutations in Philadelphia chromosome-positive (Ph+) leukemia, including the T315I mutation. It's particularly important because it offers a treatment option for patients whose leukemia has relapsed or become resistant to other therapies. Ponatinib's ability to target these resistant mutations can allow more patients to then proceed to a potentially curative bone marrow transplant.

What is allogeneic hematopoietic cell transplantation (HCT), and why is it considered in treating this type of leukemia?

Allogeneic hematopoietic cell transplantation (HCT), also known as bone marrow transplant, is a procedure where a patient receives healthy blood-forming stem cells from a donor. In the context of Philadelphia chromosome-positive (Ph+) leukemia, HCT offers the potential for a cure by replacing the patient's diseased bone marrow with healthy donor cells. It's a significant treatment option, especially for those who have not responded well to other therapies, because it can provide long-term remission.

What does 'salvage therapy' mean in the context of leukemia treatment?

Salvage therapy, in this context, refers to the use of treatments like low-dose ponatinib to control relapsed or refractory Philadelphia chromosome-positive (Ph+) leukemia. The goal is to reduce the disease to allow a patient to then receive allogeneic hematopoietic cell transplantation (HCT). It is crucial because it aims to rescue patients who have exhausted other treatment options, providing a bridge to potentially life-saving HCT.

What is the T315I mutation, and why is it important in leukemia?

The T315I mutation is a specific mutation within the tyrosine kinase domain that confers resistance to many tyrosine kinase inhibitors (TKIs) used to treat Philadelphia chromosome-positive (Ph+) leukemia. Its presence often leads to treatment failure, making it a significant challenge in managing the disease. Ponatinib is effective against the T315I mutation, making it a valuable option for patients with this mutation.

What are tyrosine kinase inhibitors (TKIs) and how do they work in treating leukemia?

Tyrosine kinase inhibitors (TKIs) are a class of drugs that target and inhibit the activity of tyrosine kinases, enzymes that play a role in cell growth and division. In Philadelphia chromosome-positive (Ph+) leukemia, the abnormal BCR-ABL tyrosine kinase drives the uncontrolled growth of leukemia cells. TKIs like ponatinib block the activity of this enzyme, thereby slowing or stopping the progression of the leukemia. Their effectiveness has significantly improved outcomes for patients, particularly in chronic phase chronic myeloid leukemia (CP-CML).