Center For International Blood & Marrow Transplant Research

MINNEAPOLIS--( BUSINESS WIRE )--Research results from NMDP℠ and CIBMTR® (Center for International Blood and Marrow Transplant Research®)—published today in the peer-reviewed Journal of Clinical Oncology —found no discernable difference in overall survival (OS) among patients with blood cancer receiving blood stem cell transplant when investigators used an unrelated donor matched at 7/8 human leukocyte antigen (HLA) markers, compared to a fully matched 8/8 unrelated donor when using a post-transplant cyclophosphamide-based (PTCy) graft-versus-host disease (GVHD) prevention strategy. Conducted by CIBMTR—a research collaboration between the Medical College of Wisconsin® and NMDP—the observational study also showed no discernable differences in GVHD-free, relapse-free survival (GRFS) for adults with blood cancers who had a 7/8 or an 8/8 unrelated donor transplant when using PTCy. The study, “ Post-Transplant Cyclophosphamide–Based Graft-Versus-Host Disease Prophylaxis Attenuates Disparity in Outcomes Between Use of Matched or Mismatched Unrelated Donors,” evaluated 10,025 adult patients at 153 U.S. transplant centers who underwent initial unrelated donor blood stem cell transplant for acute leukemia or myelodysplastic syndromes from 2017–2021 and reported outcomes to CIBMTR. The most clinically significant outcome observed showed that when compared to 8/8 unrelated donor transplant using PTCy, 7/8 unrelated donor transplant with PTCy had similar OS (hazard ratio [HR], 0.96 [95% Confidence Interval, 0.823-1.11]; P = .60) and similar GRFS (HR, 0.90 [0.79-1.02]; P = .1). Previously established methods to prevent GVHD involved use of immunosuppressants called calcineurin inhibitors, yet outcomes were traditionally worse with mismatched donors. In this study, when compared to 8/8 unrelated donor transplant using traditional calcineurin-inhibitor GVHD prevention methods, OS was improved after 8/8 PTCy transplant (HR, 0.88 [0.80-0.96]; P = .004) and similar with 7/8 PTCy transplant (HR, 0.92 [95% CI, 0.80 to 1.05]; P = .2062). GRFS was improved after 8/8 (HR, 0.61 [0.57-0.66]; P < .0001) or 7/8 PTCy transplant (HR, 0.68 [0.60-0.76]; P < .0001). Investigators showed adoption of novel treatment modality PTCy can eliminate disparities in survival, greatly reduce GVHD risk, and expand safe and effective transplant access to people for whom a matched unrelated donor is unavailable, which disproportionally impacts patients who are racially and ethnically diverse. “ These data confirm we are at the forefront of transplant practice change. As our global population becomes more diverse and geographically disparate, patients facing blood cancer are increasingly looking for a safe and effective method to increase their survival and longevity,” Brian Shaffer, MD, co-first author; bone marrow transplant specialist & cellular therapist, Memorial Sloan Kettering Cancer Center, said. “ Achieving recognition from one the most prestigious, peer-reviewed medical publications demonstrates the credibility of these data and builds the evidential case for using mismatched unrelated donors to expand access to blood stem cell therapy for all patients.” In the U.S., patients with blood cancers from Asian/Pacific Islander backgrounds currently have a 47% chance of finding a fully matched, 8/8 unrelated donor on the NMDP Registry℠ of potential volunteer donors. However, those odds increase to 92% when searching for a 7/8 match or greater. African American and Black patients’ chances jump from 29% with an 8/8 match, to as high as 84% with a 7/8 match—while patients with Latino / Hispanic or non-Hispanic White ancestry improve their odds from 48% (8/8) to 90% (7/8) and 79% (8/8) to 99% (7/8), respectively. 1 “ This novel approach transforms the landscape for physicians, researchers, patients and families, expanding the definition of a ‘suitable donor,’” said Steven Devine, MD, chief medical officer, NMDP and senior scientific director, CIBMTR . “ Not only does achieving significantly greater outcomes give patients another chance at life, it breaks down the barriers to transplant for all patients, regardless of their ancestry.” These results are part of NMDP’s ongoing research commitment to expand access to cell therapy to all patients and close the donor availability gap. NMDP’s network of transplant centers, many of which participate in CIBMTR trials, are collaborating to bring new research to light that is challenging previously established blood stem cell transplantation science. NMDP’s Donor for All initiative includes several research efforts, including this published study, that aim to establish a new, safe and effective approach for using mismatched unrelated donor transplants in the U.S. and abroad. Operational data from NMDP-facilitated transplants show 60% year-over-year growth in mismatched unrelated donor transplant—the majority of which are from unrelated donors matched at 7/8 HLA markers, indicating how the U.S. medical community is seeking other safe and effective, evidence-based methods to prolong patients’ ability to survive and thrive. 2 “ This is scientific evidence showing we are addressing an unmet need,” Dr. Devine added. CIBMTR presented findings from this study at the American Society for Transplantation and Cellular Therapy’s scientific congress, Tandem 2024 , in February 2024. About CIBMTR® CIBMTR® (Center for International Blood and Marrow Transplant Research®) is a nonprofit research collaboration between NMDP℠, in Minneapolis, and the Medical College of Wisconsin, in Milwaukee. CIBMTR collaborates with the global scientific community to increase survival and enrich quality of life for patients. CIBMTR facilitates critical observational and interventional research through scientific and statistical expertise, a large network of centers, and a unique database of long-term clinical data for more than 675,000 people who have received hematopoietic cell transplantation and other cellular therapies. Learn more at cibmtr.org . About NMDP ℠ At NMDP℠, we believe each of us holds the key to curing blood cancers and disorders. As a global nonprofit leader in cell therapy, NMDP creates essential connections between researchers and supporters to inspire action and accelerate innovation to find life-saving cures. With the help of blood stem cell donors from the world’s most diverse registry and our extensive network of transplant partners, physicians and caregivers, we’re expanding access to treatment so that every patient can receive their life-saving cell therapy. NMDP. Find cures. Save lives. Learn more at nmdp.org . 1 Chowdhury AS, Maiers M, Spellman SR, Deshpande T, Bolon YT, Devine SM. Existence of HLA-mismatched unrelated donors closes the gap in donor availability regardless of recipient ancestry. Transplant Cell Ther . 2023:29(11):686.e1-686.e8. doi: 10.1016/j.jtct.2023.08.014 . 2 Internal data on file, 2021-2023.

Highlights include a 2024 Best of ASCO® oral abstract on unrelated donor blood stem cell transplantation, late-breaking oral abstract on combination sotorasib for patients with a treatment-resistant tumor mutation called KRAS G12C, and monotherapies for people with advanced cancers LOS ANGELES--(BUSINESS WIRE)-- World-renowned physicians and researchers from City of Hope®, one of the largest cancer research and treatment organizations in the United States, will present new data and offer expert perspectives on leading-edge cancer research and treatments in development at the 2024 ASCO Annual Meeting, which will take place in Chicago from May 31 to June 4. Highlights include the following: 2024 Best of ASCO® program: New data on mismatched unrelated donor peripheral blood stem cell transplantation Late-breaking data on the phase 3 CodeBreaK 300 trial Glofitamab monotherapy for patients with advanced mantle cell lymphoma An oral inhibitor monotherapy for people with advanced kidney cancer Interventions for older adults with cancer who traditionally have not had access to supportive care treatments that could improve quality of life About 40,000 oncology professionals will attend the meeting, themed “The Art and Science of Cancer Care: From Comfort to Cure.” City of Hope experts will have 64 presentations, including oral sessions, clinical science symposiums, education discussions and the following late-breaking abstract: Overall survival (OS) of phase 3 CodeBreaK 300 study of sotorasib plus panitumumab (soto+pani) versus investigator’s choice of therapy for KRAS G12C-mutated metastatic colorectal cancer (mCRC) Presenter: Marwan G. Fakih, M.D., City of Hope medical oncologist Presentation Time and Location: Monday, June 3, 1:21 p.m. CT, Arie Crown Theater Other noteworthy City of Hope abstracts: Expanding access to stem cell transplants: Not a perfect match, yet perhaps just as good Presentation Time and Location: Friday, May 31, 3:45 p.m. CT, S100bc Access to potentially lifesaving stem cell transplants traditionally has been limited due to the need for a donor whose stem cells match the recipient’s genes. However, Monzr M. Al Malki, M.D., City of Hope hematologist-oncologist, co-led a multicenter phase 2 trial (NCT04904588) sponsored by NMDP and conducted via CIBMTR (Center for International Blood and Marrow Transplant Research) that demonstrates encouraging overall survival one year after older patients with advanced blood cancers received “HLA-mismatched unrelated donor peripheral blood stem cell transplantation,” meaning the donor stem cells originated from someone who was not a relative and not a complete HLA match. While the 13-site ongoing trial includes adults and children, this interim analysis that will be presented as an oral abstract presentation evaluated the first 70 older adult patients with advanced blood cancers who received this type of transplant and underwent both reduced intensity conditioning and post-transplant cyclophosphamide, which is a method used to prevent a potentially life-threatening side effect called graft-versus-host disease (GVHD). City of Hope has performed over 19,000 transplants, is one of the nation’s leading transplant programs and is at the forefront of using transplants to treat older adults with blood cancers. Overall survival at one year post-stem cell transplant was 79%. Rates of GVHD and other complications were comparable to those of recipients who received HLA-matched donor stem cells, suggesting HLA-mismatched unrelated donor peripheral blood stem cell transplantation may one day be used more widely, expanding access to the lifesaving therapy. “This data, supported by the National Marrow Donor Program, is promising and is the reason why I continue to work to expand access to stem cell transplantation for older patients, underrepresented people and patients with blood cancers,” Dr. Al Malki said. The adult cohorts of this trial recruitment have closed with more than 200 patients in follow-up. The pediatric arm of the trial is ongoing. Bispecific antibody demonstrates sustained response rates in lymphoma patients who received and rejected previous treatments Presentation Time and Location: Saturday, June 1, 5:24 p.m. CT, S100bc In an updated efficacy and safety phase 1/2 trial (NCT03075696), bispecific antibody glofitamab continues to demonstrate compelling response rates with a fixed-duration treatment among a group of mantle cell lymphoma patients who have nearly exhausted approved treatment options due to aggressive disease. The researchers evaluated close to 60 participants who had received two or more previous treatments — the majority of which had stopped responding to treatment before they enrolled in this study. Patients received about 7.4 months of glofitamab. Landmark analyses indicated that most patients with a complete response at the end of treatment were alive without disease progression 15 months after the end of treatment. The median duration of complete response was 12.6 months, and median progression-free survival was 8.6 months. “People with mantle cell lymphoma have a rare form of non-Hodgkin lymphoma that is currently incurable, so it is encouraging to see people who were told by others that there is not much more to be done live months and even years longer with this treatment,” said Tycel Phillips, M.D., City of Hope hematologist-oncologist and presenting author. “We at City of Hope continuously develop leading-edge treatments for people diagnosed with lymphoma and other blood cancers.” Early safety and clinical efficacy data on monotherapy DFF332 in advanced kidney cancer patients Presentation Time and Location: Saturday, June 1, from 8:30 a.m. CT, S100bc People with an advanced kidney cancer called clear cell renal cell carcinoma (ccRCC) appeared to experience positive medicinal effects from the targeted therapy pill DFF332 while encountering manageable side effects in an ongoing first-in-human phase 1/1B multicenter trial (CDFF332A12101, NCT04895748). Sumanta “Monty” Pal, M.D., a City of Hope medical oncologist, will share preliminary data on 40 advanced kidney cancer patients who took DFF332, an oral hypoxia-inducible factor (HIF)-2α inhibitor that has been shown in preclinical models to be effective in reducing ccRCC tumors. All study participants had received at least one other type of therapy before joining the clinical trial. At data cutoff, 16 patients continued to be treated, while 19 patients (48%) stopped receiving the treatment due to disease progression. The most common side effects were fatigue (33%), anemia (30%), increased blood cholesterol and constipation (15%). No extreme adverse effects have been observed so far. At cutoff, 18 patients (45%) had stable disease and two patients (5%) achieved partial response. “While the study is still in process, so far, we have seen a promising safety pro monotherapy DFF332 with indications of clinical activity,” Dr. Pal said. “We are conducting analysis on how the medicine moves within the body as well as collecting biomarker data that will be shared at our ASCO presentation.” Using telehealth to improve access to supportive care services for older adults with cancer living in lower-resourced communities Presentation Time and Location: Saturday, June 1, at 1:39 p.m. CT, S102 A study at City of Hope | Antelope Valley found that telehealth can be leveraged to offer older adults living in high-poverty, lower resourced communities with a specialized evaluation developed at City of Hope called a geriatric assessment, which identifies supportive care services older adults with cancer need to manage vulnerabilities, better define care goals and improve quality of life. This quality improvement study included 251 participants who were 65 years or older with newly diagnosed or advanced cancer. These patients underwent a baseline geriatric assessment and most had initial visits with a geriatric nurse practitioner – 197 via televideo and 45 via telephone. The assessment identified vulnerabilities in 209 patients and, after review, the nurse practitioner made 460 necessary referrals for supportive care services – 86% of which were implemented. The most common referrals were to pharmacy (177), social work (142), occupational therapy (76) and physical therapy (48). More than 92% of patients reported they were satisfied with the telehealth services that resulted in supportive care interventions for older adults. “In my experience, if you don’t do a geriatric assessment, the patient pays for it later,” said Tanyanika Phillips, M.D., M.P.H., presenting author and City of Hope medical oncologist and hematologist in Antelope Valley, California. Phillips noted that if care is prescribed without accurate health information, the result could be serious side effects or even hospitalization, which could lead not only to more costly care but also cancer treatment outcomes that are not the best for patients. “In a general visit, patients often will say they feel well and are fine because they’re incentivized to answer in the affirmative and move forward with treatment," Phillips added. “Place this same patient in a different environment where they are answering geriatric assessment questions, and they may be more forthcoming and detailed about their lifestyle and abilities. This candor will help physicians prescribe the most appropriate care for that individual based on their circumstances.” Providing supportive care interventions via telehealth based on a geriatric assessment is shown to improve daily functioning, happiness and quality of life Presentation Time and Location: Sunday, June 2, from 11:54 a.m. CT, S100bc A randomized trial at a Brazilian cancer center and under the guidance of City of Hope’s internationally renowned cancer and aging expert William Dale, M.D., Ph.D., found that older adults with metastatic cancer reported experiencing significant improvements in the performance of daily activities, emotional well-being and quality of life after receiving a telehealth-based geriatric assessment (GA) that resulted in supportive care interventions. This data extends City of Hope’s prior work in GA-guided supportive care beyond the borders of the United States. “Our studies continue to prove that patients and families win when care teams ask older adults with cancer the right questions at the outset to guide care. This is true even when telehealth is used in a low-resource environment. Guidance from a GA can change care choices and improve outcomes — all without making cancer therapy less effective. It’s a form of precision medicine: more appropriate supportive care interventions, better daily functioning, higher quality of life and the same great cancer care results. It’s a winning formula for patients, families, providers and the health system,” said Dr. Dale, senior author of the study, City of Hope’s George Tsai Family Chair in Geriatric Oncology and director of City of Hope’s Center for Cancer and Aging. Dr. Dale is the recipient of this year’s B.J. Kennedy Geriatric Oncology Award, which honors geriatric oncologists who have demonstrated outstanding leadership and achievement in the field of geriatric oncology. Additional highlights include the following award-winning poster abstract and three education sessions: “A phase I/II trial of palbociclib, pembrolizumab, and endocrine therapy for patients with HR+/HER2- locally advanced or metastatic breast cancer (MBC): Clinical outcomes and stool microbial profiling”* Presentation Time: Sunday, June 2, from 9 a.m. to noon CT Presenter: Alexis LeVee, M.D., City of Hope Hematology & Medical Oncology Chief Fellow *Conquer Cancer, the ASCO Foundation, awarded Dr. LeVee a 2024 ASCO Annual Meeting Merit Award, which supports students and trainees who are first authors on abstracts selected for presentation. “Evidence Evaluating Cannabis’ Efficacy Across the Cancer Care Continuum” Monday, June 3, 8:30 a.m. CT Session title: Evidence-Based Integrative Oncology: Guideline Insights for Comprehensive Care Presenter: Richard T. Lee, M.D., City of Hope clinical professor, Supportive and Integrative Medicine Program; Cherng Family Director's Chair for the Center for Integrative Oncology “Tissue-Based Molecular Testing and the Role of Artificial Intelligence” Monday, June 3, 10 a.m. CT Session title: Should I Order the Test? Expanding the Array of Emerging Diagnostics in Breast Cancer Presenter: Daniel Schmolze, M.D., City of Hope associate clinical professor, Department of Pathology “What Is Variant Histology Renal Cell Cancer and What Are the Available Treatment Options?” Monday, June 3, 3:15 p.m. CT Session title: Managing Variant Histologies in Urothelial and Renal Cell Cancers Presenter: Sumanta Kumar Pal, M.D., City of Hope professor, Department of Medical Oncology & Therapeutics Research About City of Hope City of Hope's mission is to make hope a reality for all touched by cancer and diabetes. Founded in 1913, City of Hope has grown into one of the largest cancer research and treatment organizations in the U.S. and one of the leading research centers for diabetes and other life-threatening illnesses. City of Hope research has been the basis for numerous breakthrough cancer medicines, as well as human synthetic insulin and monoclonal antibodies. With an independent, National Cancer Institute-designated comprehensive cancer center at its core, City of Hope brings a uniquely integrated model to patients spanning cancer care, research and development, academics and training, and innovation initiatives. City of Hope’s growing national system includes its Los Angeles campus, a network of clinical care locations across Southern California, a new cancer center in Orange County, California, and cancer treatment centers and outpatient facilities in the Atlanta, Chicago and Phoenix areas. City of Hope’s affiliated group of organizations includes Translational Genomics Research Institute and AccessHope™. For more information about City of Hope, follow us on Facebook, X, YouTube, Instagram and LinkedIn.

Adults with blood cancers receiving peripheral blood stem cell transplant exhibited excellent survival rates and low incidence of life-threatening graft-versus-host-disease Additional NMDP presentation on population-level access barriers to bone marrow transplant MINNEAPOLIS--(BUSINESS WIRE)-- NMDPSM, a global nonprofit leader in cell therapy, and the CIBMTR® (Center for International Blood and Marrow Transplant Research®), announced that interim results from the ACCESS trial will be presented as an oral abstract on Friday, May 31 at the American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, Ill., demonstrating that adults with hematologic malignancies who received peripheral blood stem cell (PBSC) transplant from HLA-mismatched unrelated donors (MMUD) followed by post-transplant cyclophosphamide (PTCy) graft-versus-host-disease (GvHD) prophylaxis exhibited a 79% overall survival, with a significant 51% GvHD-free, relapse-free survival (GRFS) probability at one-year post transplant. Notably, ASCO also selected this abstract to be presented at its 2024 Best of ASCO program in July. “We want more patients to survive and thrive — results from ACCESS to-date have shown positive, significant transplant outcomes and good quality of life for patients,” said Monzr M. Al Malki, M.D., Associate Professor, Department of Hematology & Hematopoietic Cell Transplantation, City of Hope; presenting author and ACCESS study co-chair. “These preliminary data provide strong support for inclusion of this novel treatment approach for patients receiving PBSC from partially matched, unrelated donors and advance current evidence demonstrating how more patients can benefit from the potentially curative therapeutic effects of transplant.” In addition to achieving very good OS and GRFS clinical endpoints, adult participants also exhibited low rates of severe acute and chronic GvHD, both at 9%. The NMDP-sponsored ACCESS trial, conducted through the CIBMTR — a research collaboration between the Medical College of Wisconsin and NMDP — enrolled 70 adult patients with blood cancers and disorders, including acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), and acute lymphoblastic leukemia (ALL), from 13 participating transplant centers, like City of Hope. All patients in this stratum received reduced intensity conditioning, most often fludarabine/melphalan (63%) or fludarabine/busulfan (20%), and PBSCs from donors matched at 5-7 of 8 HLA loci, predominantly at the 7/8 match level (67%). Notably, half of patients enrolled in the trial were people of color. “Historically, barriers in access to transplant have been inhibited by two factors: the low availability of matched, related sibling donors; and the substantial variance of matched, unrelated donor availability on international registries, particularly for patients with diverse ancestry — many of whom already face significant challenges,” said study co-author Steven M. Devine, M.D., Chief Medical Officer, NMDP; Senior Scientific Director, CIBMTR. “Our research findings advance our ability to offer more options to patients without a fully matched donor, many of whom are ethnically diverse and have been underserved in receiving potentially life-saving cell therapy.” Addressing Equitable Care: NMDP-led Donor for All Research Traditionally, finding an available matched, unrelated donor on international registries has been limited for patients with ethnically diverse ancestry – from as low as 29% for Black or African American patients and 48% for Latino and Hispanic patients versus 79% for non-Hispanic Whites. NMDP’s network of transplant centers, many of which participate in CIBMTR trials, are bringing new research to light that is challenging previously established stem cell transplantation science. ACCESS builds upon findings from the first “Donor for All” trial, a groundbreaking NMDP-sponsored 15-MMUD study, which showed that PTCy was effective in decreasing risk for GvHD in adults with hematologic malignancies receiving bone marrow (BM) transplants from MMUD. Similar to ACCESS, 15-MMUD patients — 48% of whom were ethnically diverse — showed a one-year overall survival (OS) rate of 76% and exhibited a low rate of moderate / severe GvHD. Enrolling now, the OPTIMIZE trial is evaluating whether a reduced dose of PTCy will safely and effectively prevent GvHD while reducing infection risk in patients with hematologic malignancies receiving PBSC HCT from MMUDs. Finally, a recent observational study by the CIBMTR presented at the 2024 Tandem Meetings, reported no discernable differences in OS or GRFS for adult patients with hematologic malignancies using MMUD HCT at an 8/8 or 7/8 HLA match level using PTCy GvHD prophylaxis — increasing the likelihood for patients of all ethnicities of finding a suitable donor to at least 84% and up to 99%. “Our Donor for All research is the foundational future upon which we are building a new platform protocol using innovative strategies for preventing and treating GvHD, decreasing risk for post-transplant relapse of hematologic malignancies, and in the future, applying MMUD transplant to cure non-malignant conditions, such as sickle cell disease,” said Dr. Devine. “Through CIBMTR, we are showing that science can solve the gap in equitable access to transplant, giving new hope to patients worldwide.” 2024 ASCO Presentation Details Oral Presentation (Abstract #6503) Friday, May 31; 2:45-5:45 p.m. CDT; Room S100bc Post-transplant cyclophosphamide-based graft-versus-host disease prophylaxis following mismatched unrelated donor peripheral blood stem cell (PBSC) transplantation Monzr M. Al Malki, M.D. Poster Discussion (Abstract #1528) Saturday, June 1; 9 a.m. – noon CDT; Hall A Identifying states for targeted alloHCT access initiatives using social vulnerability, physician density, and unmet need Samantha Watters, MPH About CIBMTR® CIBMTR® (Center for International Blood and Marrow Transplant Research®) is a nonprofit research collaboration between NMDPSM, in Minneapolis, and the Medical College of Wisconsin, in Milwaukee. CIBMTR collaborates with the global scientific community to increase survival and enrich quality of life for patients. CIBMTR facilitates critical observational and interventional research through scientific and statistical expertise, a large network of centers, and a unique database of long-term clinical data for more than 675,000 people who have received hematopoietic cell transplantation and other cellular therapies. Learn more at cibmtr.org. About NMDPSM At NMDPSM, we believe each of us holds the key to curing blood cancers and disorders. As a global nonprofit leader in cell therapy, NMDP creates essential connections between researchers and supporters to inspire action and accelerate innovation to find life-saving cures. With the help of blood stem cell donors from the world’s most diverse registry and our extensive network of transplant partners, physicians and caregivers, we’re expanding access to treatment so that every patient can receive their life-saving cell therapy. NMDP. Find cures. Save lives. Learn more at nmdp.org.