The Weill Cornell Medicine and NewYork-Presbyterian Bone Marrow and Stem Cell Transplant Program researchers and physicians made a vast impact at this year’s American Society of Hematology (ASH) Annual Meeting and Exposition, an educational gathering of over 25,000 clinicians and scientists from around the world who are working to conquer blood disease.

Our team was involved in several research updates presented at the meeting, including one that was selected as “Best of ASH” by the meeting committee. Research presented at ASH 2017 helps to deepen and advance overall understanding of the different types of patients and blood diseases best suited for transplant, ways to make transplantation safer and more effective, as well as how bone marrow and stem cell transplant can improve overall survival for patients. Here are some highlights of our research.

Drs. Melissa Cushing, Director of Transfusion Medicine and Cellular Therapy at Weill Cornell Medicine (WCM)/NewYork-Presbyterian (NYP) and Ruchika Goel, Assistant Medical Director of Transfusion Medicine and Cellular Therapy at Weill Cornell Medicine/NewYork-Presbyterian, shared research findings conducted with the WCM/NYP BMT team focusing on a new protocol for red blood cell transfusions in stem cell transplant patients.

Blood transfusions can be life-saving, but can also be associated with adverse events and worse outcomes due to exposure to the blood donor’s foreign proteins, and potentially undetected infections. One way to decrease unnecessary transfusions is to use a restrictive transfusion threshold (hemoglobin level). The hemoglobin level reflects the ability of red blood cells to carry oxygen to the body’s organs. In 2015, our Bone Marrow and Stem Cell Transplant Program adopted a more restrictive red blood cell transfusion threshold. This research demonstrated the efficacy and safety of reducing unnecessary transfusions without affecting patient mortality or re-admission rates.

To view the full study abstract, click here.

Dr. Usama Gergis, hematologist and medical oncologist at the Weill Cornell Medicine and NewYork-Presbyterian Bone Marrow and Stem Cell Transplant Program, presented clinical trial preliminary results indicating that using cord blood units as adoptive immunotherapy for patients with refractory acute myeloid leukemia (AML) is a safe, well-tolerated and effective treatment. This trial involved chemotherapy followed by the infusion of umbilical cord blood units selected based on certain genetic characteristics of the patient. To date, 14 patients with very treatment-resistant AML have participated. Eight patients had objective hematologic responses and eight patients went on to receive bone marrow transplant. Five out of the 14 patients enrolled in the trial achieved complete remission.

To view the full study abstract, click here.

Dr. Koen Van Besien, Director of the Bone Marrow and Stem Cell Transplant Program at Weill Cornell Medicine and NewYork-Presbyterian, shared extremely positive results from the JULIET clinical trial demonstrating how CAR-T cell therapy is revolutionizing the treatment of hematological malignancies. The trial showed that Tisagenlecleucel (also known by the brand name Kymriah), a CAR T-cell therapy, produced high response rates in a cohort of patients with relapsed or refractory diffuse large b-cell lymphoma (DLBCL) who had received multiple lines of prior therapy. In the JULIET trial, two thirds of patients responded to this treatment and 90% of the responses were sustained at six months. The high response rate is especially significant because these patients’ disease had not responded to previous treatments. Additionally, this research was chosen as a “Best of ASH” update based on the trial’s groundbreaking results.

To view the full study abstract, click here.

Dr. Tsiporah Shore, Associate Director of the Bone Marrow and Stem Cell Transplant Program at Weill Cornell Medicine and NewYork-Presbyterian, presented initial results from a three part-phased research project designed to eventually cure sickle cell disease. This trial is the first-of-its-kind to use plerixafor, a receptor antagonist that mobilizes hematopoietic stem cells from the bone marrow to the peripheral blood for collection, in order to use these stem cells in conjunction with autologous transplant for patients with sickle cell disease. This is an exciting study that assesses the effectiveness of mobilizing and collecting stem cells in patients with sickle cell disease so that these cells can be modified and used to transplant normal healthy cells back into the same patient in an effort to cure their disease. The first step in this process is to collect enough stem cells. The second step involves making the genetic changes necessary to fix the abnormal cells. After that, the third phase comprises transplanting them back into the patient.

To view the full study abstract, click here.

Dr. Alexandra Gomez, hematology and oncology fellow in the Division of Hematology and Medical Oncology at Weill Cornell Medicine and NewYork-Presbyterian, presented findings from a phase II clinical trial evaluating a novel conditioning strategy for autologous transplant in multiple myeloma. This trial was designed to establish the efficacy of bendamustine, an anti-cancer chemotherapy drug known as an alkylating agent, in combination with melphalan, also an alkylating agent chemotherapy drug, in patients undergoing an initial autologous stem cell transplant. In a small group of patients, results showed that this combination was well tolerated and indicated promising efficacy. More than half of the patients had deep myeloma responses, and the time to relapse after transplant appeared longer than with the standard melphalan alone.

To view the full study, click here.

Dr. Lizamarie Bachier, former hematology and medical oncology fellow at Weill Cornell Medicine and NewYork-Presbyterian, presented findings from retrospective clinical research she conducted here with faculty in the BMT Program. This research analyzed patients who had undergone allogeneic stem cell transplants to determine risk factors for Epstein-Barr Virus (EBV) and Post-Transplant Lymphoproliferative Disorder (PTLD). The risk factors for EBV and PTLD were found to be undergoing an HLA-mismatched transplant and being above the age of 60. Her findings have led to prevention strategies in high-risk patients.

To view the full study, click here.

Dr. Sebastian Mayer, hematologist and medical oncologist in the Bone Marrow and Stem Cell Transplant Program at Weill Cornell Medicine and NewYork-Presbyterian, shared promising clinical research updates at ASH 2017 that may lead to new ways to identify, intervene, and ultimately overcome high-risk acute graft versus host disease after bone marrow transplant.

Amphiregulin (AREG), a protein involved in cellular growth signaling, can be elevated in patients with acute Graft versus Host Disease (GVHD), indicating a poor steroid response. In the study, results showed that regardless of the clinical stage of acute GVHD, an elevated AREG blood level was associated with a worse outcome. This test can help identify patients with the need for more aggressive therapy early on.

To view the full study, click here.

We are incredibly proud of our research that was presented at this year’s ASH meeting, and of our clinicians and researchers who work tirelessly every day to improve the lives of those with blood diseases.