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HCT Practice Guidelines for MDS

Explore Cell Therapy podcast season 2, episode 7

This two-part Explore Cell Therapy podcast episode features Zachariah DeFilipp, MD, and Andrew Brunner, MD, discussing their work and findings on a committee of the American Society for Transplantation and Cellular Therapy (ASTCT) to publish an evidence-based review on guidelines for the use of allogeneic hematopoietic cell transplantation (HCT) in the management and treatment of myelodysplastic syndromes (MDS) from the perspective of both transplant and hematology/oncology physicians.

Listen as they discuss the goals of the guidelines review, the advances in and challenges of managing MDS, and the best practices for HCT for patients with different presentations of the disease. You'll gain insights into MDS treatment, the latest HCT research related to MDS, and advances in MDS risk stratification to assist both hematology/oncology practices and transplant centers in improving HCT access, especially for older patients with MDS.

5 key takeaways from HCT Practice Guidelines for MDS

  1. The evidence-based review committee of the ASTCT set out to evaluate the data and develop a consensus on HCT guidelines and how to clinically approach patients with MDS. MDS is a heterogeneous disease, and patients have different presentations, limitations, and quality of life. The major challenge is finding the right intervention for each patient at the appropriate time.
  2. Recent clinical trial data solidify evidence that patients with high-risk MDS features undergoing transplantation earlier in their treatment course may be beneficial. With the evolution from IPSS to IPSS-R and now IPSS-M, risk stratification is moving more patients to high-risk categories, making HCT more valuable as the only potentially curative option. Two recent prospective trials found beneficial outcomes for middle-aged to older MDS patients in particular, showcasing the importance of HCT in older patients.
  3. Patients with high-risk features need to be evaluated early for transplantation, which can promote informed decision-making. These guidelines present the optimal application of HCT for MDS patients given the spectrum of disease, and are helpful for the broader medical community, patients, and transplant and hematology/oncology physicians at all levels of experience with MDS patients.
  4. Potential physician biases or misperceptions can limit patients' access to HCT, especially those with high-risk mutations and older patients. Though HCT risks are being addressed in new ways, access remains challenging if patients are not informed by their providers.
  5. Coordinated care between hematology/oncology physicians and transplant teams can be crucial for access to treatment options and optimal patient care. Referring physicians can ease the burden for patients and help provide well-rounded solutions to barriers. Early collaboration between teams is critical in planning patient care, especially when complications arise, such as disease relapse.

This episode's expert guests 

Zachariah DeFilipp, MD
Massachusetts General Hospital
Director, BMT Medical Research
Assistant Professor of Medicine, Harvard Medical School 

Dr. DeFilipp is actively involved in translational and clinical research initiatives to improve outcomes for patients undergoing allogeneic HCT. His research has primarily focused on developing approaches to preventing and treating graft-versus-host disease. His research efforts have explored improving transplant outcomes for high-risk leukemia by incorporating targeted agents pre-and post-transplant and examining late effects among long-term transplant survivors. 

Andrew Brunner, MD
Massachusetts General Hospital
Assistant in Medicine, Division of Hematology/Oncology
Assistant Professor, Harvard Medical School

Dr. Brunner treats patients with MDS, acute myeloid leukemia (AML), and other cancers of the blood and bone marrow. His research focuses on developing novel therapeutic strategies (typically phase 1 and 2 clinical trials) for patients with MDS and AML. He has been involved in studies exploring unique targets in these cancers, including mutations in the spliceosome or targets expressed on immune effector cells. In addition, he studies MDS patient outcomes, why certain patients respond to treatment better than others, and which treatment responses are more meaningful to patients. He seeks to discover novel therapies in MDS which improve survival and patient quality of life. 

Access all Explore Cell Therapy episodes

Listen to Explore Cell Therapy on Spotify

Read articles or access trials discussed in this episode

Access resources for physicians

HCT Consultation Timing Guidelines
The HCT consultation timing guidelines from the NMDP and ASTCT identify appropriate referral timing for allogeneic or autologous HCT based on a patient's disease characteristics.

Transplant Center Directory for hematology/oncology physicians
This searchable database provides details on the transplant centers in the NMDP, such as center contact information, number of transplants by cell source, patient survival information and more.

Evolution of HCT Self-Paced Learning Course
This five-part self-paced learning course will help you better understand the current state of the science for HCT and cell therapy by following the evolution of HCT and addressing common misconceptions.

Explore resources for your patients

NMDP Jason Carter Clinical Trials Search and Support Program
The Clinical Trials Search and Support Program helps patients find and join clinical trials through one-on-one support, an easy-to-navigate website, and financial assistance to help patients pay for travel costs to participate in a clinical trial.

NMDP Patient Support Center
The NMDP Patient Support Center provides support, information and resources for transplant patients, caregivers and families. All our programs and resources are free.