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Clinical

Karen M. Winkfield, MD, PhD; Narjust Florez, MD, FASCO; Gilberto de Lima Lopes Junior, MD, MBA, FASCO; Coral Olazagasti, MD; Vonetta M. Williams, MD, PhD

 Fighting Disparities and Saving Lives: An Exploration of Challenges and Solutions in Cancer Care

INC-22-TTD0622 - /clinical/bone-marrow-and-sct

In this episode of Targeted Talks, Alfred L. Garfall, MD, director of Autologous Hematopoietic Stem Cell Transplantation and section chief of Myeloma and Hematology-Oncology at Penn Medicine, discusses the phase 2 BMT CTN 1902 trial (NCT05032820) of idecabtagene vicleucel (ide-cel; Abecma), a study focused on patients with multiple myeloma who had a suboptimal response to first-line therapy, including autologous hematopoietic cell transplant and lenalidomide (Revlimid) maintenance.

Presented at the 2025 Transplant and Cellular Therapy Meetings, the topline findings revealed that ide-cel, an anti-BCMA chimeric antigen receptor (CAR) T-cell therapy, demonstrated remarkable efficacy in this challenging patient population. At the 6-month mark, 63% of evaluable patients (n = 38) achieved a complete response (CR), with many responses occurring “quite early,” according to Garfall.

Additionally, 87% of patients achieved minimal residual disease (MRD) negativity, a critical marker for long-term remission, with 95% of these patients reaching MRD-negative status at some point during the study period. These results are particularly encouraging, as only 1 patient experienced disease progression at a median follow-up of 10.7 months.

For safety, cytokine release syndrome (CRS) occurred in 81.6% of patients, though the majority of cases were mild, with grade 1 CRS seen in 68.4% of patients and grade 2 CRS seen in 13.2%. The median time to CRS onset was just 1 day, with resolution typically occurring within 2.5 days.

No cases of immune effector cell­–associated neurotoxicity syndrome (ICANS) were reported. Additionally, infections were observed in 26.32% of patients, with most being bacterial or viral in nature and occurring within the first 100 days post-infusion.

“This is the first study that utilized CAR T cells in the BMT CTN…It was a gratifying experience to run a CAR T-cell trial in the BMT CTN to demonstrate that this network can expand to these new cellular therapies, especially if they are somehow paired or combined with bone marrow transplant or stem cell transplant, which is its conventional focus,” says Garfall.

While the final analysis, including 12-month follow-up data and ancillary studies, is still pending, the preliminary results suggest that ide-cel could offer a viable pathway for patients to continue their standard-of-care regimen.

Garfall AL, Pasquini MC, Bai L, et al. Phase II multicenter trial of idecabtagene vicleucel (ide-cel) followed by lenalidomide maintenance for multiple myeloma patients with sub-optimal response after an upfront autologous hematopoietic cell transplantation: top line results from the BMT CTN 1902 clinical trial. Presented at: 2025 Transplant and Cellular Therapy Meetings; February 12-15, 2025; Honolulu, HI. Abstract LBA-1.

In this episode of Targeted Talks, Alfred L. Garfall, discusses the phase 2 BMT CTN 1902 trial of idecabtagene vicleucel in patients with multiple myeloma.

BMT CTN 1902 in Myeloma: Key Findings With Garfall

During a live event, Catherine J. Lee, MD, MS, discussed the mechanism of action of approved therapies for chronic graft-vs-host disease and reasons to select one over others. 

Disease Site and Access Factor into Selection of GVHD Therapy

The impact of physical and nutritional rehabilitation post bone marrow transplant in patients with hematologic malignancies will be determined in the REBOOT trial.

As BMT Use Expands, Rehabilitation Gains Focus to Improve Outcomes 

During a live event, Catherine J. Lee, MD, MS, discussed the efficacy and safety results of various trials of approved agents for treating chronic graft-vs-host disease.

Lee Evaluates Chronic GVHD Therapies for Depth and Durability of Response

During a live event, Catherine J. Lee, MD, MS, discussed the results of the AGAVE-201 trial.

Axatilimab Leads to Strong Responses in Heavily Pretreated cGVHD

Orca-T improved 1-year survival without severe graft-vs-host disease compared with standard transplant in the Precision-T study.

Orca-T Improves cGVHD-Free Survival Across Hematologic Malignancies

Popat Covers Itacitinib and GVHD Prevention Trial Results

Uday R. Popat, MD, professor of medicine in the Department of Stem Cell Transplantation and Cellular Therapy, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center, discusses promising findings from a phase 2 trial evaluating the JAK inhibitor itacitinib (INCB039110) combined with post-transplant cyclophosphamide (PTCy) and tacrolimus for graft-vs-host disease (GVHD) prevention in patients undergoing a myeloablative fractionated busulfan regimen.

According to findings presented at the 2025 Transplant and Cellular Therapy Meetings, a total of 31 patients with a median age of 62 (range, 25-69) years were enrolled. Ten (32%) patients had acute myeloid leukemia, 10 (32%) had myelofibrosis, 6 (19%) had myelodysplastic syndrome, and 1 each had chronic lymphocytic leukemia, lymphoma, chronic myeloid leukemia, myeloma, and acute lymphoblastic leukemia.

At day 100, the cumulative incidence of grade 2, 3, or 4 acute GVHD was 26% (10%-42%), and was seen in 8 patients. Moderate to severe chronic GVHD was observed in 4.5% of patients, and there were no deaths related to GVHD.

At a median follow up of 18 months, the 1-year overall survival, progression-free survival, GVHD-free relapse free survival, non-relapse mortality, and relapse rates were 81%, 81%, 70%, 16%, and 3.2% respectively. These results suggest effective GVHD control without compromising anti-leukemic efficacy, particularly in older patients.

“This is a trial where we enrolled patients between 18 and 70, so this is a reasonably older group of patients. Our median age was 62, ranging between 25 and 69. What we saw here was 1-year survival of 80%, which was pretty decent.”

Popat UR, Alousi AM, Bassett RL, et al. JAK inhibitor itacitinib with PTCY and tacrolimus to prevent GVHD in a myeloablative fractionated busulfan regimen: A phase 2 trial. Presented at: 2025 Transplant and Cellular Therapy Meetings; February 12-15, 2025; Honolulu, HI. Abstract 37.



Videos

Uday R. Popat, MD, discusses promising findings from a phase 2 trial evaluating the JAK inhibitor itacitinib combined with post-transplant cyclophosphamide and tacrolimus for graft-vs-host disease prevention.

News

Choosing Itacitinib for GVHD Prophylaxis: Insights From Popat

Uday R. Popat, MD, professor of medicine in the Department of Stem Cell Transplantation and Cellular Therapy, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center, discusses why he and his fellow researchers chose to evaluate the JAK inhibitor itacitinib (INCB039110) over other JAK inhibitors for graft-vs-host disease (GVHD) prophylaxis in a phase 2 study that was presented on at the 

2025 Transplant and Cellular Therapy Meetings

In the phase 2 trial, experts sought to assess the use of the JAK inhibitor itacitinib when combined with post-transplant cyclophosphamide (PTCy) and tacrolimus to prevent GVHD in patients undergoing a myeloablative fractionated busulfan regimen.

According to Popat, itacitinib was selected for evaluation in this study due to its lack of hematopoiesis or myelosuppression seen in in vitro models. During transplant, donor grafts require stem cell engraftment and blood count recovery, making low myelotoxicity critical.

Because of itacitinib’s minimal myelotoxic profile, experts believe that the agent offers significant potential value, aligning with the need for safe, effective agents in transplantation. As a result, itacitinib was chosen for evaluation in this phase 2 study.

So, we looked at itacitinib. Firstly, because in in vitro models, it did not cause any suppression of hematopoiesis or myelosuppression. Now, in transplant [and] during transplant, we have given donor graft, and we are allowing the stem cells to engraft and blood counts to recover.

 So, [it is] an agent with low myelotoxicity [that] has a significant potential value. [This] is why we chose this particular agent.

Popat UR, Alousi AM, Bassett RL, et al. Jak inhibitor itacitinib with ptcy and tacrolimus to prevent gvhd in a myeloablative fractionated busulfan regimen: A phase 2 trial. Presented at: 2025 Transplant and Cellular Therapy Meetings; February 12-15, 2025; Honolulu, HI. Abstract 37.

Uday R. Popat, MD, discusses the rationale behind evaluating itacitinib over other JAK inhibitors for graft-vs-host disease prophylaxis in a phase 2 study.


Conference News

Preventing GVHD With Itacitinib in Stem Cell Transplants

Uday R. Popat, MD, professor of medicine in the Department of Stem Cell Transplantation and Cellular Therapy, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center, discusses a phase 2 trial exploring the use of the JAK inhibitor itacitinib (INCB039110) combined with post-transplant cyclophosphamide (PTCy) and tacrolimus to prevent graft-vs-host disease (GVHD) in patients undergoing a myeloablative fractionated busulfan regimen.

This innovative approach aims to reduce non-relapse mortality and improve transplant outcomes by targeting GVHD.

The main reasons for stem cell transplant failure are relapse of the original disease and treatment-related mortality, or non-relapse mortality, due to the transplant. To improve outcomes, the best thing—or the only thing—to do would be to reduce non-relapse mortality and reduce the relapse rate.

 In this abstract, we aimed to reduce non-relapse mortality by reducing the incidence and severity of graft-vs-host disease in patients undergoing allogeneic transplantation. So we have, systematically over a period of time, developed consecutive protocols to achieve this. First, we lengthened the duration of the regimen, [which we call] a fractionated busulfan regimen. Instead of giving it over a 4-day period, we give it over a 3-week period.

 What this allows us to do is reduce the toxicity of the treatment and enhance non-relapse mortality. It also allows us to deliver a myeloablative dose of chemotherapy to older patients, which in turn reduces the relapse rate.

 In addition to fractionating the busulfan, we added post-transplant cyclophosphamide to reduce graft-vs-host disease. In this study, we took the next step by adding itacitinib, which is a JAK inhibitor. It is known that JAK inhibitors are useful in the prevention and treatment of graft-vs-host disease. We hypothesized that a JAK inhibitor might also be useful in the prevention of graft-vs-host disease, reducing non-relapse mortality and further improving our treatment regimen

Uday R. Popat, MD, discusses a phase 2 trial exploring the use of itacitinib combined with post-transplant cyclophosphamide and tacrolimus to prevent graft-vs-host disease in patients undergoing a myeloablative fractionated busulfan regimen. 


Advancing Stem Cell Therapy and Equity in Patient Care

Jeff Auletta, MD, senior vice president of health equity at the National Marrow Donor Program and chief scientific officer of the Center for International Blood and Marrow Transplant Research, discusses his takeaways from the 

Specifically, Auletta highlights the emerging science in stem cell transplants and cell therapies, including treatments like chimeric antigen receptor (CAR) T-cell therapy. He also discussed topics presented at the meeting that went beyond scientific advancements, including important discussions on advocacy. He highlights that these topics emphasize the social vulnerability of patients and the need for systemic change to increase access to life-saving therapies.

According to Auletta, addressing these disparities requires a collective, ecosystem-wide effort in order to ensure better representation and access for vulnerable patients in stem cell transplants and cell therapies.

 Overall, I think that the Tandem Meetings were fantastic, with regards to the emerging science, both in terms of stem cell transplants and cell therapies—those therapies that either are directed against infection in the patient or malignant disease like CAR T cells. So, it was a great meeting in terms of hearing the advancing science and collaborating with folks, but also the advocacy in terms of, as an ecosystem, understanding the social vulnerability of our patients and wanting to make a system-level change in order to get more patients to transplant and these life-saving therapies.

And that is really going to take an ecosystem response, and that's what we want to do—is really drive forth the message that, you know, for lots of reasons, these patients have a vulnerability and aren't well represented in terms of stem cell transplant or cell therapy. And we, as an ecosystem, really have a moral responsibility to address these things, and that's what we're doing as a whole, in terms of an ecosystem.

Jeff Auletta, MD, discusses his key takeaways from the 2025 Transplant and Cellular Therapy Meetings.


Ladbury Discusses Advancements in Total Marrow Irradiation

Colton Ladbury, MD, a radiation oncologist and assistant clinical professor in the Department of Radiation Oncology at City of Hope, discusses radiation therapy for hematologic malignancies, total marrow irradiation (TMI), and findings that were presented during the American Society for Radiation Oncology (ASTRO) Annual Meeting.

 Radiation has been used historically as part of the conditioning regimen for patients undergoing transplants for hematologic malignancies. That has been done for decades. It is very non-targeted, called total body irradiation for a reason as it treats the entire body.

 At City of Hope, somewhere around 2006, we started a different technique called total marrow irradiation where they thought, [if a patient has] leukemia, we do not need to treat the entire body. We probably need to just focus on either the bone marrow plus or minus the lymph nodes. Around that time, there were advances in radiation technology that made it possible to focus on those parts and spare other parts of the body.

 That was something that we have been doing more of since we can decrease toxicities by not giving radiation to the parts of the body that do not necessarily need it, and by not having as much toxicity, we may be able to go to a higher dose of radiation, which may have better efficacy against leukemia.

This was all theoretical at first. We previously looked at a small group of patients who have been treated with TMI to look at their toxicities, but what this abstract was trying to do is double that number of patients and have a little bit more long-term follow-up to look at if we are reducing toxicity compared with historical controls, and what are the unmet needs that we still need to look for in terms of long-term toxicities?

Colton Ladbury, MD, discusses radiation therapy for hematologic malignancies, total marrow irradiation, and findings that were presented during the American Society for Radiation Oncology Annual Meeting.


Sallman on Eprenetapopt and Azacitidine in TP53-Mutant MDS/AML

David Sallman, MD, Moffitt Cancer Center, discusses the study design, objectives, and rationale behind using eprenetapopt and azacitidine together for the treatment of in patients with 

-mutant myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) post-allogeneic stem cell transplantation.

 We had a clinical trial actually published in [T

], which was a maintenance clinical trial of azacitidine with eprenetapopt following stem cell transplantation. The results showed quite impressive outcomes, potentially supporting the development of a future phase 3 study.

 One big question was, could the analysis of TP53 MRD by deep sequencing, in this case, by duplex technology, further and better predict outcomes? [Currently,] there is very little data known in the transplant world about the impact of TP53 MRD.

 We [collected] bone marrow samples at multiple time points: before transplantation, after transplantation but prior to initiating therapy, and at standard disease assessment intervals—namely at day 100, 6 months, and 12 months. Using these samples, we evaluated the role of TP53 MRD in predicting whether patients would experience relapse within the context of this maintenance trial.

-mutant patients represent, really, the molecular subset with the poorest of outcomes across myelodysplastic syndromes and acute myeloid leukemia, plastic syndrome and acute myeloid leukemia. Long-term survival in these patients is only about 20%, regardless of age or fitness. This has sparked some debate about whether these patients should undergo stem cell transplantation. I believe they should, although there are cases where borderline performance status may raise legitimate questions about proceeding with the procedure.

 Novel strategies to reduce relapse risk in this group are urgently needed. Eprenetapopt is a first-in-class TP53 reactivator that has shown significant benefit when combined with azacitidine in several phase 1/2 clinical trials. Unfortunately, a pivotal phase 3 trial, while demonstrating improved complete remission rates, did not meet statistical significance. We are still awaiting the formal presentation and publication of these data.

 [Given these challenges], we conducted a maintenance trial using a slightly reduced dose from earlier studies but maintaining the same combination of eprenetapopt and azacitidine. This time, the focus was on using the regimen as a post-transplant maintenance strategy rather than as an initial treatment to improve patient outcomes.

David Sallman, MD, discusses using eprenetapopt and azacitidine in TP53-mutant myelodysplastic syndrome and acute myeloid leukemia post-allogeneic stem cell transplantation.

Current Treatment Protocols for Ph+ ALL Treatment 

Josep Maria Ribera, MD, PhD, Catalan Institute of Oncology, discusses the current standard treatment protocols for Philadelphia chromosome (Ph)-positive acute lymphoblastic leukemia (ALL) and the rationale behind them. He then delves into the primary benefits of combining chemotherapy with transplant in this space.

 We have to distinguish between assistential protocols and clinical trials. For assistential protocols in the real world, we are still using first- and second-generation TKI combined with low-intensity chemotherapy, and we follow these patients by [measurable residual disease (MRD)]. Then, the MRD level at 3 months after the initiation of therapy is the perfect moment to indicate the need for allogeneic stem cell transplantation.

Now, clinical trials use third-generation TKIs [and] use immunotherapy, and the panorama has completely changed. Outstanding responses, potent MRD negativity, deep MRD negativity—and then the indication for transplantation in these clinical trials remains marginal. It is important to distinguish what we are dealing with: clinical trials or assistential trials.

The benefit of TKIs and chemotherapy is to provide a high grade of response. The complete cytologic response is over 100%, and the molecular response is about 50% so this is an improvement. This limits the indication of transplantation to the patients who are MRD positive, but we can spare transplantation in those who are MRD negative. This is what occurs in real life.

Now, the advent of chemo[therapy]-free trials, immunotherapy-based trials, third–generation-based trials, improve the response and improve the quality of the response, and all of it is achieved with low-grade toxicity, so that they are feasible at all age groups. This is important for [older patients] in whom Ph-positive ALL is observed in half of ALL patients. It is important that now, the progress has been not only in efficacy, but also in tolerability and availability.

Josep Maria Ribera, MD, PhD, discusses the current standard treatment protocols for Philadelphia chromosome-positive acute lymphoblastic leukemia and the rationale behind them.


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