Hany Elmariah

All Publications

• Improved Patient-Reported Outcomes With Post-Transplant Cyclophosphamide: A Quality-of-Life Evaluation and 2-Year Outcomes of BMT CTN 1703. Journal of clinical oncology : official journal of the American Society of Clinical Oncology Holtan, S. G., Bolaños-Meade, J., Al Malki, M. M., Wu, J., Kitko, C. L., Reshef, R., Rezvani, A. R., Shaffer, B. C., Solh, M. M., Yao, J. M., Runaas, L., Elmariah, H., Larkin, K. T., El Jurdi, N., Gooptu, M., Loren, A. W., Hall, A. C., Alousi, A. M., Jamy, O., Clark, W., Kean, L., Bhatt, A. S., Perales, M. A., Applegate, K., Efebera, Y. A., Leifer, E., Jones, R. J., Horowitz, M. M., Mattila, D., Saber, W., Hamadani, M., Martens, M. J. 2025: JCO2400921

  Abstract

  The BMT CTN 1703 phase III trial confirmed that graft-versus-host disease (GVHD) prophylaxis with post-transplantation cyclophosphamide (PTCy), tacrolimus (Tac), and mycophenolate mofetil (MMF) results in superior GVHD-free, relapse-free survival (GRFS) compared with Tac/methotrexate (MTX) prophylaxis. This companion study assesses the effect of these regimens on patient-reported outcomes (PROs). Using the Lee Chronic GVHD Symptom Score and PROMIS subscales (physical function, GI symptoms, social role satisfaction) as primary end points and hemorrhagic cystitis symptoms and Lee subscales as secondary end points, responses from English and Spanish speakers were analyzed at baseline and days 100, 180, and 365 after transplant. PRO scores were compared between the arms using inverse probability weighted-independent estimating equation models. The PTCy arm had significantly lower scores on the Lee Chronic GVHD Symptom Scale (P = .01), indicating lower GVHD symptom burden. Lee Scale nutrition and mouth subscores were also better in the PTCy arm compared with the Tac/MTX arm (P < .01 for both). Older participants (age >65 years) reported better Lee Scale psychological subscores than younger participants (P = .003). No significant differences were identified in hemorrhagic cystitis or in the PROMIS subscales between treatment arms. The updated clinical end points at 2 years for the parent trial confirmed that PTCy/Tac/MMF maintained a significant advantage over Tac/MTX in GRFS (42.4% v 28.8%, P = .001). In addition to improved GRFS, patients randomly assigned to the PTCy arm reported lower symptom burden during the first year after transplant.

  View details for DOI 10.1200/JCO.24.00921

  View details for PubMedID 39752608

• LARGE-SCALE TCR DEEP SEQUENCING REVEALS A T CELL DIVERSITY BOTTLENECK WITH POST-TRANSPLANT CYCLOPHOSPHAMIDE WITH IMPLICATIONS FOR EFFICACY AND TOXICITY: RESULTS OF THE BMT-CTN 1801 STUDY Kean, L., Siegel, S., Schmalz, J., Bien, S., DeWolf, S., Dong, J., Scheffey, D., Sanders, C., Robins, H., Applegate, K., Bar, M., Chhabra, S., Choi, S., Clark, W., Das, S., Jenq, R., Jones, R., Levine, J., Logan, B., Murthy, H., Rashidi, A., Riches, M., Saber, W., Sandhu, K., Sung, A., Larkin, K., Al Malki, M., Gooptu, M., Elmariah, H., Alousi, A., Runaas, L., Shaffer, B., Rezvani, A., El Jurdi, N., Loren, A., Horowitz, M., Bolanos-Meade, J., Holtan, S., Bhatt, A., Perales, M. SPRINGERNATURE. 2024: 12-13

  View details for Web of Science ID 001365375000003

• Large-Scale Post-Transplant TCR Deep Sequencing Reveals a Major T Cell Diversity Bottleneck with Post-Transplant Cyclophosphamide with Implications for Both Efficacy and Toxicity: Results of the BMT CTN 1801 Study Kean, L., Siegel, S. J., Schmalz, J. T., Bien, S. A., Scheffey, D., Sanders, C., Robins, H., Applegate, K., Bar, M., Chhabra, S., Choi, S. W., Clark, W., Das, S. R., Jenq, R. R., Jones, R. J., Levine, J., Logan, B. R., Murthy, H. S., Rashidi, A., Riches, M. L., Saber, W., Sandhu, K. S., Sung, A. D., Larkin, K., Al Malki, M. M., Gooptu, M., Elmariah, H., Alousi, A., Runaas, L., Shaffer, B. C., Rezvani, A. R., El Jurdi, N., Loren, A., Horowitz, M. M., Bolanos-Meade, J., Holtan, S. G., Bhatt, A. S., Perales, M., DeWolf, S. AMER SOC HEMATOLOGY. 2023

  View details for DOI 10.1182/blood-2023-188032

  View details for Web of Science ID 001159306701232

• Patient-Reported Outcomes of BMT CTN 1703: A Randomized Phase III Study for Gvhd Prophylaxis - a Quality of Life Evaluation Holtan, S. G., Martens, M. J., Al Malki, M. M., Efebera, Y., Kitko, C. L., Reshef, R., Rezvani, A. R., Shaffer, B. C., Solh, M. M., Yao, J. M., Runaas, L., Elmariah, H., Larkin, K. T., El Jurdi, N. H., Gooptu, M., Loren, A. W., Hall, A. C., Alousi, A., Jamy, O., Clark, W. AMER SOC HEMATOLOGY. 2023

  View details for DOI 10.1182/blood-2023-187859

  View details for Web of Science ID 001159306703166

• Post-Transplantation Cyclophosphamide-Based Graft-versus-Host Disease Prophylaxis. The New England journal of medicine Bolaños-Meade, J., Hamadani, M., Wu, J., Al Malki, M. M., Martens, M. J., Runaas, L., Elmariah, H., Rezvani, A. R., Gooptu, M., Larkin, K. T., Shaffer, B. C., El Jurdi, N., Loren, A. W., Solh, M., Hall, A. C., Alousi, A. M., Jamy, O. H., Perales, M. A., Yao, J. M., Applegate, K., Bhatt, A. S., Kean, L. S., Efebera, Y. A., Reshef, R., Clark, W., DiFronzo, N. L., Leifer, E., Horowitz, M. M., Jones, R. J., Holtan, S. G. 2023; 388 (25): 2338-2348

  Abstract

  In patients undergoing allogeneic hematopoietic stem-cell transplantation (HSCT), a calcineurin inhibitor plus methotrexate has been a standard prophylaxis against graft-versus-host disease (GVHD). A phase 2 study indicated the potential superiority of a post-transplantation regimen of cyclophosphamide, tacrolimus, and mycophenolate mofetil.In a phase 3 trial, we randomly assigned adults with hematologic cancers in a 1:1 ratio to receive cyclophosphamide-tacrolimus-mycophenolate mofetil (experimental prophylaxis) or tacrolimus-methotrexate (standard prophylaxis). The patients underwent HSCT from an HLA-matched related donor or a matched or 7/8 mismatched (i.e., mismatched at only one of the HLA-A, HLA-B, HLA-C, and HLA-DRB1 loci) unrelated donor, after reduced-intensity conditioning. The primary end point was GVHD-free, relapse-free survival at 1 year, assessed in a time-to-event analysis, with events defined as grade III or IV acute GVHD, chronic GVHD warranting systemic immunosuppression, disease relapse or progression, and death from any cause.In a multivariate Cox regression analysis, GVHD-free, relapse-free survival was significantly more common among the 214 patients in the experimental-prophylaxis group than among the 217 patients in the standard-prophylaxis group (hazard ratio for grade III or IV acute GVHD, chronic GVHD, disease relapse or progression, or death, 0.64; 95% confidence interval [CI], 0.49 to 0.83; P = 0.001). At 1 year, the adjusted GVHD-free, relapse-free survival was 52.7% (95% CI, 45.8 to 59.2) with experimental prophylaxis and 34.9% (95% CI, 28.6 to 41.3) with standard prophylaxis. Patients in the experimental-prophylaxis group appeared to have less severe acute or chronic GVHD and a higher incidence of immunosuppression-free survival at 1 year. Overall and disease-free survival, relapse, transplantation-related death, and engraftment did not differ substantially between the groups.Among patients undergoing allogeneic HLA-matched HSCT with reduced-intensity conditioning, GVHD-free, relapse-free survival at 1 year was significantly more common among those who received cyclophosphamide-tacrolimus-mycophenolate mofetil than among those who received tacrolimus-methotrexate. (Funded by the National Heart, Lung, and Blood Institute and others; BMT CTN 1703 ClinicalTrials.gov number, NCT03959241.).

  View details for DOI 10.1056/NEJMoa2215943

  View details for PubMedID 37342922

• NCCN Guidelines Insights: Hematopoietic Cell Transplantation, Version 3.2022. Journal of the National Comprehensive Cancer Network : JNCCN Saad, A., Loren, A., Bolanos-Meade, J., Chen, G., Couriel, D., Di Stasi, A., El-Jawahri, A., Elmariah, H., Farag, S., Gundabolu, K., Gutman, J., Ho, V., Hoeg, R., Horwitz, M., Hsu, J., Kassim, A., Kharfan Dabaja, M., Magenau, J., Martin, T., Mielcarek, M., Moreira, J., Nakamura, R., Nieto, Y., Ninos, C., Oliai, C., Patel, S., Randolph, B., Schroeder, M., Tzachanis, D., Varshavsky-Yanovsky, A. N., Vusirikala, M., Algieri, F., Pluchino, L. A. 2023; 21 (2): 108-115

  Abstract

  The NCCN Guidelines for Hematopoietic Cell Transplantation (HCT) provide an evidence- and consensus-based approach for the use of autologous and allogeneic HCT in the management of malignant diseases in adult patients. HCT is a potentially curative treatment option for patients with certain types of malignancies; however, recurrent malignancy and transplant-related complications often limit the long-term survival of HCT recipients. The purpose of these guidelines is to provide guidance regarding aspects of HCT, including pretransplant recipient evaluation, hematopoietic cell mobilization, and treatment of graft-versus-host disease-a major complication of allogeneic HCT-to enable the patient and clinician to assess management options in the context of an individual patient's condition. These NCCN Guidelines Insights provide a summary of the important recent updates to the NCCN Guidelines for HCT, including the incorporation of a newly developed section on the Principles of Conditioning for HCT.

  View details for DOI 10.6004/jnccn.2023.0007

  View details for PubMedID 36791762

• Post-Transplant Cyclophosphamide, Tacrolimus, and Mycophenolate Mofetil As the New Standard for Graft-Versus-Host Disease (GVHD) Prophylaxis in Reduced Intensity Conditioning: Results from Phase III BMT CTN 1703 Holtan, S. G., Hamadani, M., Wu, J., Al Malki, M. M., Runaas, L., Elmariah, H., Rezvani, A. R., Gooptu, M., Larkin, K. T., Shaffer, B. C., El Jurdi, N. H., Loren, A. W., Solh, M., Hall, A. C., Alousi, A. M., Jamy, O. H., Perales, M., Yao, J. M., Applegate, K., Bhatt, A. S., Kean, L. S., Efebera, Y. A., Kitko, C., Reshef, R., Clark, W., DiFronzo, N. L., Henderson, L., Jones, R. J., Liefer, E., Martens, M. J., Horowitz, M. M., Bolanos-Meade, J. AMER SOC HEMATOLOGY. 2022: 14-16

  View details for DOI 10.1182/blood-2022-171463

  View details for Web of Science ID 000897957100005

• Early cytopenias and infections after standard of care idecabtagene vicleucel in relapsed or refractory multiple myeloma. Blood advances Logue, J. M., Peres, L. C., Hashmi, H., Colin-Leitzinger, C., Shrewsbury, A. M., Hosoya, H., Gonzalez, R., Copponex, C., Kottra, K. H., Hovanky, V., Sahaf, B., Patil, S., Lazaryan, A., Jain, M. D., Baluch, A., Klinkova, O., Bejanyan, N., Faramand, R. G., Elmariah, H., Khimani, F., Davila, M. L., Mishra, A., Blue, B., Grajales-Cruz, A. F., Castaneda Puglianini, O., Liu, H., Nishihori, T., Freeman, C. L., Brayer, J., Shain, K. H., Baz, R., Locke, F. L., Alsina, M., Sidana, S., Hansen, D. K. 2022

  Abstract

  Idecabtagene vicleucel (ide-cel) was FDA approved in March 2021 for the treatment of relapsed/refractory multiple myeloma (RRMM) after 4 lines of therapy. On the KarMMa trial, grade ≥3 cytopenias and infections were common. We sought to characterize cytopenias and infections within 100 days after ide-cel in the standard of care (SOC) setting. This multi-center retrospective study included 52 patients who received SOC ide-cel; 47 reached day 90 follow-up. Data was censored at day 100. Grade ≥3 cytopenia was present among 65% of patients at day 30 and 40% of patients at day 90. Granulocyte colony stimulating factor (G-CSF) was administered to 88%, packed red blood cell (pRBC) transfusions to 63%, platelet transfusions to 42%, thrombopoietin (TPO) agonists to 21%, intravenous immunoglobulin (IVIG) to 13%, and CD34+ stem cell boosts to 8%. At day 100, 19% and 13% of patients had ongoing use of TPO agonists and G-CSF, respectively. Infections occurred in 54% of patients and were grade ≥3 in 23%. Earlier infections in the first 30 days were typically bacterial (68%) and severe (50%). Later infections between days 31 - 100 were 50% bacterial and 42% viral; only 13% were grade ≥3. On univariate analysis, high pre-CAR-T marrow myeloma burden (>/= 50%), circulating plasma cells at pre-lymphodepletion (LD), and grade ≥3 anemia at pre-LD were associated with grade ≥3 cytopenia at both days 30 and 90. Longer time from last bridging treatment to LD was the only significant risk factor for infection.

  View details for DOI 10.1182/bloodadvances.2022008320

  View details for PubMedID 35939783

• MI-Immune/1801: Lessons from an Ongoing, Multi-Center Trial Involving Biospecimen Collection for Prospective Microbiome and Immune Profiling in Patients Undergoing Reduced Intensity Conditioning Allogeneic HCT Brooks, E., Spahn, A., Waldvogel, S., Howard, A., Bar, M., Bratrude, B., Chhabra, S., Clark, W., Das, S., Horowitz, M. M., Jenq, R. R., Jones, R. J., Levine, J. E., Logan, B. R., Murthy, H. S., Rashidi, A., Riches, M. L., Riwes, M., Sandhu, K. S., Sung, A. D., Al Malki, M. M., Runaas, L., Elmariah, H., Rezvani, A. R., Gooptu, M., Bolanos-Meade, J., Holtan, S. G., Saber, W., Hamadani, M., Kean, L. S., Perales, M., Bhatt, A. S. AMER SOC HEMATOLOGY. 2021

  View details for DOI 10.1182/blood-2021-149313

  View details for Web of Science ID 000736413904028

• Umbilical cord blood or HLA-haploidentical transplantation: Real world outcomes vs randomized trial outcomes. Transplantation and cellular therapy O'Donnell, P. V., Brunstein, C. G., Fuchs, E. J., Zhang, M. J., Allbee-Johnson, M., Antin, J. H., Leifer, E. S., Elmariah, H., Grunwald, M. R., Hashmi, H., Horowitz, M. M., Magenau, J. M., Majhail, N., Milano, F., Morris, L. E., Rezvani, A. R., McGuirk, J. P., Jones, R. J., Eapen, M. 2021

  Abstract

  Randomized clinical trials offer the highest quality data for modifying clinical practice. Results of a phase III randomized trial of non-myeloablative transplantation for adults with high- risk hematologic malignancies with two umbilical cord blood (UCB) units (n=183) or HLA-haploidentical relative bone marrow (Haplo-BM) (n=154) revealed 2-year progression-free survival (PFS) of 41% and 35% after Haplo-BM and two-unit UCB transplantation, respectively (p=0.41); overall survival was 57% and 46%, respectively (p=0.04), BMT CTN 1101; NCT01597778.We sought to examine the generalizability of BMT CTN 1101 to a contemporaneous cohort beyond the trial's pre-specified 2-year outcomes. All transplantation occurred between June 2012 and June 2018 in the United States. We hypothesized that the results of a rigorous phase III randomized trial will be generalizable. Changes in graft selection for HLA-haploidentical relative transplantation during the trial period allowed comparison of outcomes after transplantation with Haplo-BM to those after haploidentical peripheral blood (Haplo-PB).The trial's broad eligibility criteria were applied to the data source of the Center for International Blood and Marrow Transplant Research to select non-trial subjects. Extended follow up of trial subjects was obtained from this data source. Three separate analyses were performed: 1) trial subjects beyond the trial's 2-year endpoint 2) comparison of trial subjects to a contemporaneous cohort of non-trial subjects (195 two-unit UCB, 358 Haplo-BM, 403 Haplo-PB) and 3) comparison of non-trial subjects by donor and graft type. Multivariate analyses were performed using Cox proportional hazards models for comparison of outcomes by treatment groups.With longer follow up of the trial cohorts, 5-year PFS (37% versus 29%, p=0.08) and overall survival (42% versus 36%, p=0.06) were not significantly different between treatment groups. We then compared the trial results to comparable real-world transplantations. Five-year overall survival after trial and non-trial two-unit UCB (36% versus 41%, p=0.48) and trial and non-trial Haplo-BM (42% versus 47%, p=0.80) transplantation were not different confirming generalizability. The randomized trial did not accrue as planned and therefore lacked the statistical power to detect a 15% difference in progression-free survival. With substantially larger numbers of non-trial Haplo-BM transplantations, 5-year survival was higher after non-trial Haplo-BM compared to trial two-unit UCB (47% versus 36%, p=0.012). Non-trial patients who received Haplo-PB transplantation had higher 5-year survival (54%) compared to trial (HR 0.76, p=0.044) and non-trial (HR 0.78, p=0.026) Haplo-BM. Similarly, survival was higher after Haplo-PB compared to trial (HR 0.57, p<0.0001) and non-trial UCB (HR 0.63, p=0.0002).When considering alternative donor low intensity conditioning regimen transplantation, a haploidentical relative is preferred. Further, PB is the preferred graft.

  View details for DOI 10.1016/j.jtct.2021.11.002

  View details for PubMedID 34775146