About

Mariam T Nawas, MD, specializes in hematopoietic cell transplantation (HCT) and cellular therapies for adults suffering from hematologic malignancies. She cares for patients with myeloid malignancies including acute myeloid leukemia, myelodysplastic syndrome, myeloproliferative neoplasms, and clonal cytopenias. Her research interests lie in better understanding and reducing HCT and cell therapy-related toxicities and symptom burden, particularly in older adults. Dr. Nawas is the Director for Quality and Data Management and the Associate Clinical Director of the Jonas Center for Cellular Therapy. She also directs the Transplant Optimization Program at the University of Chicago, a geriatric-assessment guided multidisciplinary clinic dedicated to evaluation and optimization of older adults undergoing HCT and cell therapy.

Research topics

• Medicine
• Internal medicine
• Surgery
• Computer Science
• Biology
• Intensive care medicine
• Oncology
• Genetics
• Gastroenterology
• Immunology

Selected publications

• Relationship between Social Drivers of Health and Clinician Adherence to Late Effects Screening Among Allogeneic HCT Survivors in the United States

  Transplantation and Cellular Therapy · 2026-02-01

  article
  PublisherDOI

• Challenging a clinical dogma with multimodal machine learning: a retrospective analysis of transplant mismatched donor selection

  Leukemia · 2026-02-09 · 2 citations

  articleOpen access
  PublisherOA PDFDOI

• Superior Long-Term Survival with Fludarabine-Melphalan RIC Regimen in Older AML/MDS Patients: A Propensity Score Analysis of CIBMTR Data

  Transplantation and Cellular Therapy · 2026-02-01

  article
  PublisherDOI

• Absolute Risk Trade-offs of Donor Age, Sex, and Graft Source in PTCy-Based Transplantation

  Blood Advances · 2026-05-20

  articleOpen access
  PublisherDOI

• Hematologic complications in patients exposed to poly-ADP ribose polymerase inhibitors

  Haematologica · 2026-01-29

  articleOpen access

  Not available.

  PublisherDOI

• Outcomes for Patients With Myeloid Neoplasms Treated With Chemotherapy Plus Venetoclax After Prior Venetoclax Therapy

  eJHaem · 2025-06-01 · 1 citations

  articleOpen access

  Background: Outcomes for patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that have progression after treatment with hypomethylating agent (HMA) and venetoclax (VEN) are poor. However, data for chemotherapy and VEN (C+VEN) therapy after prior treatment with HMA+VEN are limited. Methods: We identified 18 patients with AML or MDS/AML who received C+VEN after prior HMA+VEN. Results: Complete remission (CR) or CR with incomplete hematologic recovery (CRi) was achieved in 7 patients (39%) and 6 patients (33%) proceeded to allogeneic hematopoietic stem cell transplantation. Conclusion: This study shows suggests that C+VEN could be a viable option in a subset of patients after HMA+VEN.

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• Assessing Quality of Life and Symptoms in Transplantation and CAR-T Recipients: Expert Panel Recommendations from the Survivorship Special Interest Group of ASTCT

  Transplantation and Cellular Therapy · 2025-07-02 · 7 citations

  reviewOpen access
  PublisherOA PDFDOI

• Superior long-term outcomes with fludarabine and melphalan reduced intensity regimen in older AML/MDS patients undergoing allogeneic stem cell transplantation: An analysis of CIBMTR data

  Blood · 2025-11-03

  articleOpen access

  Abstract Introduction: Reduced intensity and non-myeloablative (RIC/NMA) conditioning regimens are routinely utilized in allogeneic hematopoietic cell transplantation (alloHCT) for older patients with AML/MDS. However, the efficacy of different conditioning regimens remains unclear. Previous work has suggested that the FM100 regimen is associated with improved long-term outcomes vs. more intense conditioning regimens (Ciurea S, et al. Blood 2020). Using a national registry, we compared outcomes across five commonly used fludarabine-based RIC/NMA regimens in a large cohort of older AML/MDS patients undergoing alloHCT. Methods: We included patients aged ≥50 years who were transplanted between 2013 and 2022 and reported to the CIBMTR registry. All included patients underwent their first alloHCT for AML or MDS using one of five fludarabine-based RIC/NMA regimens: fludarabine/melphalan 100 mg/m² (FM100) or 140 mg/m² (FM140), fludarabine with 2 days of busulfan (FB2), fludarabine/cyclophosphamide/2 gy TBI (FCT), or fludarabine/2 gy TBI (FT) with any graft-versus-host disease (GVHD) prophylaxis regimen. Patients who received haploidentical transplants or ex vivo T cell-depleted grafts were excluded from the analysis. To account for multiple comparisons, the false discovery rate was controlled using the Benjamini-Hochberg method. To reduce treatment allocation bias, outcomes were compared using propensity score inverse probability weighting (PS-IPW). Results: A total of 11,731 patients from 183 centers were analyzed, including FB2 (n=4,571), FM100 (n=1,666), FM140 (n=4,242), FCT (n=786), and FT (n=466). The median age was 66 years (range 50-83). Overall, 55% had HCT-CI > 2, and 51% had KPS < 90%. For the entire cohort, 9% had AML in ≥ 3rd CR or with active disease at transplant, while 9% had MDS with high/very high IPSS-R scores. Donor types included MSD (27%), MUD (60%), MMUD (7%), other related donors (2%), unrelated with unknow matching status (4%). Post-transplant cyclophosphamide for GVHD prophylaxis was administered in 15% of cases. With a median follow-up of 60 months, the adjusted 3-year OS was 53% for FM100, 53% for FM140, 48% for FB2, 47% for FCT, and 38% for FT (p < 0.0001). The 3-year GVHD-free, relapse-free survival (GRFS) was 15%, 19%, 16%, 14%, and 8%, respectively (p < 0.0001). Multivariable analysis showed that the FCT regimen was significantly associated with worse early (≤ 6 months) OS (HR 1.58, p = 0.014) and late (> 6 months) OS (HR 1.24, p = 0.011) compared to FB2. Conversely, both FM100 (HR 0.77, p < 0.0001) and FM140 (HR 0.77, p < 0.0001) were associated with better late OS than FB2. Additionally, FM100 and FM140 demonstrated higher OS in pairwise comparisons with FCT and FT regimens (p < 0.0001). Due to a significant interaction between conditioning regimens and transplant year, the disease-free survival (DFS) analysis was stratified into three transplant periods. For patients transplanted between 2019-2022, FM100 (HR 0.70, p < 0.0001), FM140 (HR 0.70, p < 0.0001), and FCT (HR 0.86, p = 0.004) showed improved DFS compared to FB2. Both FM100 and FM140 also exhibited significantly better DFS than FCT and FT, with no notable difference between FM100 and FM140. Similar trends were observed for GRFS, with FM100 and FM140 associated with improved late GRFS compared to other regimens. The survival benefit of FM regimens was primarily driven by significantly lower relapse rates compared to the other regimens, with no significant difference in relapse rates between FM100 and FM140. However, higher early transplant-related mortality (TRM) was noted in FM regimens, though they did not significantly affect TRM beyond 6 months post-transplant. No significant differences were observed in the risk of chronic or grade 2–4 acute GVHD among these groups. PS-IPW analyses demonstrated similar results, with better survival and lower relapse rates for FM regimens compared to all other RIC regimens. Conclusion: This large-scale analysis demonstrates that FM regimens, particularly FM100 and FM140, provide superior long-term survival and significantly lower relapse rates in older AML/MDS patients undergoing alloHCT. PS-IPW analyses confirmed these benefits despite early TRM risks. These results strongly support the adoption of FM regimens as the preferred conditioning approach to optimize outcomes in this population.

  PublisherOA PDFDOI

• Factors associated with survival after allogeneic transplantation for myeloid neoplasms harboring <i>TP53</i> mutations

  Blood Advances · 2025-03-14 · 11 citations

  articleOpen access

  ABSTRACT: Allogeneic hematopoietic stem cell transplant (alloHCT) is considered for all patients with myeloid neoplasms (MNs) harboring TP53 mutations (TP53mut). The aim of this international study across 7 transplant centers in the United States and Australia was to identify factors associated with improved post-alloHCT survival. Of 134 TP53mut MN cases who underwent alloHCT, 80% harbored complex karyotype; 94% of TP53 variants were localized to the DNA-binding domain (DBD). Most common comutations were ASXL1 (7%), TET2 (7%), and DNMT3A (6%). Median post-HCT survival was 1.03 years, and overall survival (OS) at 1 year, 2 years, and 3 years was 51.4%, 35.1%, and 25.1%, respectively. A total of 103 cases (76.9%) met the International Consensus Classification (ICC) criteria for MN with mutated TP53 (referred to as ICC-defined TP53mut MN hereafter). The 3-year OS of ICC-defined TP53mut was significantly shorter compared with that of other TP53mut MNs (3-year OS, 16.9% vs 54.9%; P = .002). ICC-defined TP53mut MNs was independently associated with inferior OS (hazard ratio [HR], 2.62; P = .019). The presence of non-DBD TP53mut only (HR, 3.40; P = .005), DNMT3A comutation (HR, 2.64; P = .016), and pre-alloHCT bone marrow blasts ≥5% (HR, 2.76; P = .006) was independently associated with inferior relapse-free survival (RFS), whereas melphalan-based conditioning was associated with superior RFS (HR, 0.52; P = .005). Combining these variables, we constructed a hierarchical model that stratified patients into low-, intermediate-, and high-risk categories with 1-year RFS of 81.3%, 31.3%, and 6.7%, respectively (P < .001). In conclusion, a subset of MN harboring TP53mut who have low blasts pre-alloHCT and received melphalan-based conditioning derived long-term benefit from alloHCT.

  PublisherOA PDFDOI

• Outcomes of patients with relapsed/refractory acute leukaemia treated with revumenib with a focus on post‐revumenib therapies

  British Journal of Haematology · 2025-10-26 · 1 citations

  articleOpen access

  Rearrangements of the lysine methyltransferase 2A gene (KMT2Ar) and mutations in nucleophosmin 1 (NPM1m) are among the most common genetic aberrations in acute leukaemia, with KMT2Ar seen in both acute myeloid leukaemia (AML) and acute lymphoblastic leukaemia (ALL) and NPM1m present in ~30% of AML cases.1 Historically, outcomes in relapsed/refractory (R/R) acute leukaemia with KMT2Ar or NPM1m have been poor, with a median overall survival of 6 months or less.2, 3 Both KMT2Ar and NPM1m acute leukaemia harbour leukaemogenic pathways dependent on aberrant transcription and differentiation blocks due to the protein menin. Revumenib, an oral selective menin inhibitor, disrupts leukaemogenesis in both KMT2Ar and NPM1m acute leukaemias.4 The phase 1/2 AUGMENT-101 trial of revumenib in patients with R/R KMT2Ar and NPM1m acute leukaemia demonstrated a complete remission (CR) or complete remission with partial haematological recovery (CRh) rate of 22.8% among patients with KMT2Ar and a CR + CRh rate of 23.4% in those with NPM1m.5, 6 While the single-agent efficacy of revumenib in the R/R setting is promising, the duration of response has been limited; the median overall survival (OS) was 8 months for patients with R/R AML with KMT2Ar and 4 months for those with NPM1m AML.5, 6 Currently, there are limited data regarding outcomes of subsequent therapies in patients already treated with revumenib. We sought to analyse the outcomes of patients with R/R acute leukaemia after receiving revumenib and the efficacy of subsequent treatment lines. Adult patients with R/R acute leukaemia treated with revumenib monotherapy at the University of Chicago between 22 January 2020 and 22 May 2025 were studied as part of a single-centre, retrospective cohort analysis. Patients were identified through the University of Chicago leukaemia registry and pharmacy records. Institutional review board approval was obtained. Diagnosis, relapse and disease status were confirmed according to the International Consensus Classification of myeloid neoplasms and acute leukaemias.7 Risk classification and response assessment for patients with AML utilized the European LeukemiaNet (ELN) 2022 criteria for intensive chemotherapy.8 Response assessments for patients with ALL and mixed-phenotype acute leukaemia (MPAL) utilized the ELN 2024 adult ALL criteria.9 Descriptive statistics were utilized for baseline patient characteristics. A response was defined as achieving a CR, CRh or CR with incomplete count recovery (CRi); overall response rate (ORR) was defined as CR + CRh + CRi. OS was estimated using the Kaplan–Meier method. We evaluated 26 patients treated with revumenib monotherapy for R/R acute leukaemia in our analysis. Twenty patients (76.9%) received revumenib as part of a clinical trial, 4 (15.4%) as part of an expanded access protocol and 2 (7.7%) as a commercial drug. Most patients (73%) had AML; the remainder had B-ALL (15%) or rare subtypes (12%). Demographic and biological characteristics are presented in Table 1. Fifteen patients (58%) received revumenib for KMT2Ar, 10 (38%) for an NPM1m and 1 (4%) for a KMT2A partial tandem duplication (PTD) (Figure S1A,B). The patient with KMT2A-PTD previously received all available standard therapies and was therefore treated with revumenib given preclinical rationale for menin inhibition.10 Twenty patients had a comprehensive molecular evaluation at initial diagnosis. Aside from KMT2Ar and NPM1m, common pathogenic mutations at diagnosis included fms-related tyrosine kinase 3-internal tandem duplication (FLT3-ITD) (5/20, 25%), neuroblastoma RAS viral oncogene homolog (NRAS) (5/20, 25%), Kirsten rat sarcoma viral oncogene homolog (KRAS) (4/20, 20%), tet methylcytosine dioxygenase 2 (TET2) (4/20, 20%) and tumor Protein P53 (TP53) (2/20, 10%) (Figure S1A). Responses to revumenib are summarized in Table 1; the ORR rate was 42% (11/26). Fifteen patients received additional treatment after revumenib (post-revumenib treatment). Of the 11 patients who did not receive post-revumenib treatment, 10 (91%) died either while taking revumenib, after cessation due to no response, or due to other complications of their leukaemia, while 1 (9%) remained on revumenib at the time of data cut-off (Figure 1B). The median OS of the 26-patient cohort from the time of revumenib initiation was 7.3 months (95% confidence interval (CI) [2.5, 14.4]). Fourteen patients had repeat molecular assessments via next-generation sequencing (NGS) at the time of no response or progression on revumenib (Figure S1C), of which 5 (36%) developed a multiple endocrine neoplasia 1 (MEN1) mutation, a known driver of resistance to revumenib.11 Of the 5 patients who developed MEN1 mutations, 3 (60%) had NPM1m and 2 (40%) had KMT2Ar. Of the 15 patients who received post-revumenib treatment, 5 (33%) had an NPM1 mutation, 9 (60%) had KMT2Ar and 1 (7%) had a KMT2A-PTD. Twelve patients (80%) received one or two lines of treatment following revumenib, and three patients (20%) received ≥3 lines of treatment (Table S1). As the first line of post-revumenib treatment, 6 (40%) received a hypomethylating agent (HMA) + venetoclax (ven), 5 (33%) received intensive chemotherapy (IC) + ven, 2 (13%) with FLT3 mutations received gilteritinib-based therapy, 1 (7%) with B-ALL received CD19-directed chimeric antigen receptor T-cell (CAR T) therapy and 1 (7%) received revumenib again after previously achieving a CR on revumenib, undergoing allogeneic haematopoietic stem cell transplant (allo-HSCT), and having another relapse of disease. Of the 26 patients, 18 (69%) received ven prior to revumenib. Of these, five (28%) achieved a response to revumenib. Nine patients (50%) went on to receive post-revumenib therapy. Seven of the nine patients (78%) were retreated with ven-containing regimens post-revumenib. Two of seven patients (29%) achieved a response to a ven-containing regimen post-revumenib. Following the first line of post-revumenib treatment, 8 patients (53%) had no response. Of these, 5 (62.5%) had KMT2Ar, 2 (25%) had NPM1 mutations and 1 (12.5%) had a KMT2A-PTD. Three patients achieved a CR while four achieved a CRi for an ORR of 47% (Table S1). Among these 7 patients who achieved a response, 3 (42.8%) received HMA + ven, 2 (28.8%) received IC + ven, 1 (14%) received gilteritinib-based therapy and 1 (14%) received CAR-T therapy. Among the seven patients with a response after the first line of therapy post-revumenib, three (43%) had KMT2Ar and four (57%) had an NPM1 mutation. Among the four patients with an NPM1 mutation who achieved a response after their first line of post-revumenib therapy, NPM1 measurable residual disease (MRD) status was assessed via NGS assay with 10−5 sensitivity. Two patients (50%) were MRD positive at assessment post-revumenib, and 2 (50%) achieved and maintained MRD-negative status. Of the two patients with MRD-positive disease, one patient switched to HMA therapy and has not had subsequent NPM1 MRD assessments at the time of data cut-off, and the other patient died prior to repeat MRD assessment. Four patients who received post-revumenib therapy had a MEN1 mutation. Two of these patients had NPM1m disease and received HMA + ven; both patients achieved a response. Two patients had KMT2Ar and received HMA + ven but did not have a response. Among the 15 patients who received post-revumenib treatment, the median OS was 7.5 months (95% CI [2.3, NA]) from the time of first post-revumenib treatment and was 8.3 months (95% CI [7.8, NA]) in the seven patients who achieved a CR/CRi to their first post-revumenib treatment (Figure 1A). Of the 15 patients, 7 (47%) received additional treatment lines beyond the first line of post-revumenib therapy; their courses are summarized in the swimmer plots (Figure 1A). Of the post-revumenib treatment cohort, four patients (27%) were still alive at the time of data cut-off (Figure 1A). Of the 15 patients who received post-revumenib therapy, 6 (40%) subsequently underwent allo-HSCT; three patients died from relapsed disease and three are still alive at the time of data cut-off. The median OS from the time of allo-HSCT was 8.8 months (95% CI [4.8, NA]). In summary, this analysis characterized the clinical outcomes of 26 patients with R/R acute leukaemia treated with revumenib. The ORR was 47% in the 15 patients who received post-revumenib therapy, with most patients receiving ven-containing regimens (n = 11) as the next line of treatment. Responses were seen in patients with KMT2A aberrations (3/10), NPM1 mutations (4/5) and MEN1 mutations (2/4), suggesting that additional therapy may be effective across the common aberrations seen after relapse/progression on revumenib. Of the 15 patients who received post-revumenib therapy, 6 were able to proceed to an allo-HSCT. Prior work has demonstrated that responses to menin inhibitors in the R/R setting are typically limited in duration and that if MRD-negative status can be achieved, patients benefit from proceeding to transplant promptly.5, 6, 12 Limitations of our analysis include the small sample size. Many patients received revumenib through a clinical trial or through an expanded access programme. Therefore, our findings may not reflect the broader population which has more limited access to such resources. Nevertheless, our study provides insight into potential therapeutic strategies for revumenib-exposed patients. To our knowledge, this is the first report describing outcomes of subsequent therapies after revumenib exposure in a real-world cohort. We found that post-revumenib therapies, particularly venetoclax-based approaches, can induce responses across mutational subsets including MEN1. In addition, consolidation with allo-HSCT is feasible in eligible patients who achieve a response. Prospective studies evaluating therapies, including other menin inhibitors, in patients already treated with revumenib will be critical to understand how to improve outcomes in this group.13 As menin inhibitors are investigated in the front-line setting, similar studies might inform combinatorial trial designs and identify effective treatments after menin inhibitor therapy.14, 15 Miles Thomas was responsible for project design, data collection, data analysis and manuscript creation. Hannah Johnston was responsible for project design, data collection, data analysis and manuscript creation. Emily Dworkin was responsible for manuscript creation. Austin Wesevich was responsible for manuscript creation. Gregory W. Roloff was responsible for manuscript creation. Caner Saygin was responsible for manuscript creation. Mariam T. Nawas was responsible for manuscript creation. Michael W. Drazer was responsible for manuscript creation. Adam S. DuVall was responsible for manuscript creation. Satyajit Kosuri was responsible for manuscript creation. Michael J. Thirman was responsible for manuscript creation. Olatoyosi Odenike was responsible for manuscript creation. Wendy Stock was responsible for manuscript creation. Richard A. Larson was responsible for manuscript creation. Rafael Madero-Marroquin was responsible for project concept and design, data collection, data analysis and manuscript creation. Anand A. Patel was responsible for project concept and design, data collection, data analysis and manuscript creation. The authors would like to thank the patients whose data are represented in the study below. Anand A. Patel is supported by the NCI Early Career Investigator Award (3P30CA014599-49S1). Miles Thomas: No conflicts of interest to disclose. Hannah Johnston: No conflicts of interest to disclose. Emily Dworkin: Honoraria from AbbVie. Austin Wesevich: Honorarium from Amgen. Gregory W. Roloff: Advisory boards for Autolus Therapeutics and Kite/Gilead. Caner Saygin: No conflicts of interest to disclose. Mariam T. Nawas: No conflicts of interest to disclose. Michael W. Drazer: Scientific advisory board for Argenx. Adam S. DuVall: Speaker for CE Concepts. Satyajit Kosuri: No conflict of interest to disclose. Michael J. Thirman: Has acted as a consultant or advisor to AbbVie, AstraZeneca, Celgene, Janssen, Pharmacyclics and Roche/Genentech. Research funding from AbbVie (Inst), Gilead Sciences, Janssen, Merck, Nurix, Pharmacyclics, Syndax and TG Therapeutics. Olatoyosi Odenike: Institutional research funding by AbbVie, Astra Zeneca, Celgene, Curis, Incyte, Shattuck Lab and K-group alpha; scientific advisory board participant for AbbVie, Celgene/BMS, Novartis, Incyte, Kymera therapeutics, Servier and Rigel; service on data safety board for Treadwell therapeutics. Wendy Stock: Advisor for Kura, Servier, Newave and Asofarma. Richard A. Larson has acted as a consultant or advisor to Ariad/Takeda, CVS/Caremark, Epizyme/Ipssen and Novartis and has received clinical research support to his institution from Astellas, Biomea, Cellectis, Daiichi Sankyo, Forty Seven/Gilead and Novartis and royalties from UpToDate. Rafael Madero-Marroquin: No conflicts of interest to disclose. Anand A. Patel: Honoraria from AbbVie, Amgen, Astellas, Jazz, Sobi, Syndax; research funding (institutional) from Pfizer, Incyte, Servier and Sumitomo. Informed consent was waived as per institutional review board approval due to the retrospective nature of the study. Those seeking to reproduce material from this manuscript should reach out to the corresponding author for permission. The data used for this study can be made available in its de-identified form at the request of the corresponding author upon reasonable request and following institutional data sharing practices. To protect the confidentiality and security of individual health records, these data will not be made publicly available. Figure S1. Table S1. Please note: The publisher is not responsible for the content or functionality of any supporting information supplied by the authors. Any queries (other than missing content) should be directed to the corresponding author for the article.

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Frequent coauthors

• Anand Patel

  12 shared

• Sergio Giralt

  Memorial Sloan Kettering Cancer Center

  12 shared

• Michael W. Drazer

  University of Chicago

  9 shared

• Miguel-Ángel Perales

  9 shared

• Wendy Stock

  University of Chicago

  8 shared

• Satyajit Kosuri

  University of Chicago

  8 shared

• Michael Scordo

  Cornell University

  8 shared

• Richard A. Larson

  University of Chicago

  7 shared