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Khera, Nandita

https://orcid.org/0000-0002-0577-3155

Mayo Clinic

Mayo Clinic

Citations 163

CPX-351 versus venetoclax plus hypomethylating agents in newly diagnosed acute myeloid leukemia: A comparative analysis of outcomes in 600 patients
Fathima, Saubia, Rokach, Lior , Ghosoun, Nour, Rezasoltani, Mahsa , Alsugair, Ali, Abdelmagid, Maymona, Al‐Kali, Aref , Begna, Kebede H. , Mangaonkar, Abhishek A. , Matin, Aasiya , Litzow, Mark R. , Patnaik, Mrinal M. , Pardanani, Animesh , Foran, James M. , Palmer, Jeanne , Punwani, Nathan , Sproat, Lisa, Khera, Nandita , Tefferi, Ayalew , Gangat, Naseema , Fathima, Saubia , McCullough, Kristen , Alkhateeb, Hassan B. , Saliba, Antoine N. , Hefazi, Mehrdad , Hogan, William J. , Shah, Meera , Badar, Talha , Murthy, Hemant S. , Yi, Cecilia Arana

Blood | December 5, 2025 | Volume: 146 | Issue: Supplement 1 | Pages: 1602-1602

10.1182/blood-2025-1602 Get It

Abstract Venetoclax (Ven) in combination with hypomethylating agents (HMA) is widely used as frontline treatment in older or unfit patients with acute myeloid leukemia (AML). Liposomal cytarabine and daunorubicin (CPX-351) is FDA-approved for the treatment of secondary AML, including therapy-related (t-AML) and AML with myelodysplasia-related changes (AML-MR). The current study aimed to provide a comprehensive comparison between the two treatment strategies to better inform clinical decisions. Methods The current retrospective analysis was performed under an institutional review board approved minimal risk protocol at Mayo Clinic (MN, AZ, FL). AML-MR gene mutations or cytogenetic abnormalities were categorized according to the International Consensus Classification (Arber et al. Blood 2022;140:1200). Treatment response was assessed according to the 2022 European LeukemiaNet (ELN) criteria (Dohner et al. Blood 2022;140:1345). Overall survival (OS) analysis was censored for allogeneic stem cell transplant (ASCT). Statistical analyses were performed using JMP Pro (Version 18.0.0). Results A total of 600 patients with newly diagnosed AML (ND-AML) treated with CPX-351 (N-112, 19%), or Ven-HMA (N=488, 81%) were included. AML subtypes included de novo (N=277, 46%), post-myelodysplastic syndrome (post-MDS, N=114,19%), post-myeloproliferative neoplasm (post-MPN, N=70, 12%), post-MDS/MPN (N=36, 6%), t-AML (N=103, 17%) and AML-MR (N=274, 46%). Compared with those treated with Ven-HMA, patients receiving CPX-351 were younger (median age 65 vs 73 years; p<0.01), more likely to be female (50% vs 38%; p=0.02), more frequently diagnosed withsecondaryAML (68% vs 51%; p<0.01), and less likely to have NPM1MUT (5% vs 12%; p=0.02) whereas ELN 2022 cytogenetic risk distribution was similar between the two (adverse risk; 43% vs 40%; p=0.5). Comparison of treatment response and survival Complete response rates with (CR) or without (CRi) count recovery were similar between CPX-351 and Ven-HMA overall (55% vs 60%) as well as specifically in patients with AML-MR (60% vs 63%), adverse karyotype (44% vs 44%), NPM1MUT (100% vs 89%), TP53MUT(32% vs 45%), IDH1MUT (60% vs 76%), IDH2MUT (63% vs 77%), SF3B1MUT(71% vs 45%), and CBLMUT (33% vs 69%) [p>0.05 in all instances]. Compared to those with CPX-351, CR/CRi rates with Ven-HMA were superior in males (60% vs. 45%; p=0.04) and in patients with de novo AML (68% vs. 50%; p=0.03), STAG2MUT (86% vs. 44%; p=0.02), or CEBPAMUT (88% vs. 50%; p=0.03); infectious complications were significantly more frequent with CPX-351 (83% vs 62%, p<0.01). At a median follow-up of 12 months (range; 1-88), 416 (69%) deaths, 179 relapses (44% in CPX-351 and 52% in Ven-HMA responders) and 149 ASCTs (53% in CPX-351 and 18% in Ven-HMA) were documented. OS rates were similar between CPX-351 and Ven-HMA, in general (median 10 vs 13 months), as well as in patients with adverse karyotype, TP53MUT, IDH1MUT, and IDH2MUT (p>0.05 in all instances). By contrast, OS was superior in Ven-HMA-treated patients with post-MDS AML (median 12 vs. 7 months; age-adjusted p=0.02) and in CPX-351-treated patients harboring SF3B1MUT(median not reached vs 14 months; age-adjusted p<0.01). In addition, a borderline-significant OS advantage for CPX-351 was apparent in patients with PTPN11MUT (13.5 vs 8 months; p=0.08). Comparison in ICC defined AML-myelodysplasia-related CR/CRi and OS rates were comparable between patients treated with CPX-351 (N=57) or Ven-HMA (N=217), with response rates of 60% vs 63% (p=0.68) and median OS of 10 vs 14 months (p=0.61), respectively. However, OS was superior with Ven-HMA in patients with post-MDS AML (15 vs 8 months; age-adjusted p=0.02) and with CPX-351 in those with SF3B1MUT (NR vs 14 months; age-adjusted p=0.02). Conclusions: The current study suggests that Ven-HMA was as effective and possibly less toxic than CPX-351 for the treatment of ND-AML, including AML-MR, despite the bias for younger and fitter patients treated with the latter regimen. Observed differences involving patients with SF3B1MUT or post-MDS AML require validation in prospective studies and provide parameters for developing treatment-specific predictive models that will be presented at the meeting.
Overall survival after post-transplant cyclophosphamide (PTCy) peripheral blood hematopoietic cell transplantation (HCT) differs by race, ethnicity, and donor
Nair, Nisha, Khera, Nandita , Hahn, Theresa , Herr, Megan M. , Holtan, Shernan G.

Blood | December 5, 2025 | Volume: 146 | Issue: Supplement 1 | Pages: 963-963

10.1182/blood-2025-963 Get It

Abstract Introduction Hispanic and non-Hispanic Black (NHB) patients have worse HCT survival compared to non-Hispanic White (NHW) patients and were less likely to utilize an unrelated HCT donor (Khera et al. Blood Adv. 2024; Hahn et al. JAMA Netw Open. 2024), but this difference has not been evaluated in the PTCy era. PTCy reduces graft-versus-host disease (GvHD) and allows for increased availability of allogeneic donors in minority patients. It is important to know the best donor source for each ethnicity. In this study, we investigated the association of race/ethnicity and donor type on overall survival (OS). Methods We performed a secondary analysis of adult patients (>19 yrs) who were selected if they had a peripheral blood allogeneic HCT from 2013-2018 and received PTCy for GVHD prophylaxis using a publicly available dataset from the Center for International Blood and Marrow Transplantation Research Program. Kaplan-Meier curves estimated 2-year OS, and differences were compared using log-rank tests. X2 tests helped identify potential confounders. Analyses were stratified by donor type and self-reported race/ethnicity. The Cox Proportional Hazards model evaluated 2-year OS using a backwards elimination method to build the final prediction model. P<0.05 was considered statistically significant. Results Of 4,031 patients included, 70% were NHW, 16% were NHB, and 13% were Hispanic. The most common donor was Haploidentical (Haplo; 64%), followed by matched unrelated donor (MUD; 19%), matched related donor (MRD; 10%), and mismatched unrelated donor 7%). Hispanic patients were younger than NHW and NHB (P<0.0001) and more were transplanted in CR (P<0.0001). Additionally, most Hispanic and NHB patients received a Haplo HCT (77 and 83%, respectively). NHW patients also received Haplo HCTs (57%) as well as MUD HCTs (24%). Most patients had acute myeloid leukemia (40%) followed by myelodysplastic syndrome (22%) and received reduced intensity conditioning (58%). MUD patients who received PTCy had the highest 2-year OS compared to other donors (65 vs 58-61%; P=0.03). Higher OS was observed in younger patients (20-39 yrs: 69% vs 60+ yrs: 53%; P<0.001) and patients with non-malignant diseases (80%) or Hodgkin lymphoma (79%) compared to patients with plasma cell (52%) and myelodysplastic disorders (55%; P<0.0001). For NHW patients, patients transplanted with a MUD had higher OS than other donor types (67 vs 58-60%; p=0.0006). In contrast, NHB transplanted with MRD, Haplo, or MMUD had the highest OS compared to MUD, although this was not statistically significant (61-66% vs 42%; p=0.4). Hispanic patients transplanted with a related donor (MRD and Haplo) had higher OS compared to patients who had an unrelated donor (MUD and MMUD; 66-67 vs 48-58%; P=0.1). Within each donor source, we tested for a racial disparity in OS. There was no difference in OS by race/ethnicity in MRD or MMUD HCTs. In Haplo HCTs, Hispanic patients had the highest OS followed by NHB and then NHW (66, 61, vs 58%; P=0.005). In MUD HCTs, NHW patients had higher OS than Hispanic or NHB patients (67, 48, vs 42%; P=0.02). Significant factors associated with OS in multivariable analyses included donor type, age, disease, year of transplant, conditioning intensity, Karnofsky Performance Score, donor/recipient CMV serostatus, and disease risk index. Results were stratified by race/ethnicity. For NHW patients, mortality risk was higher for HCT patients receiving a MRD, Haplo, or MMUD compared to a MUD (HR=1.25-1.39; P=0.001). In contrast, NHB patients receiving a MUD had a higher risk of mortality compared to a Haplo, MRD, or MMUD (HR=1.67; P=0.06). In Hispanic patients, MUDs and MMUDs had a higher mortality risk than MRD or Haplo HCT (HR=1.48; P=0.07). Conclusion OS differed by race/ethnicity and donor type. Our findings should be validated by expanding the analysis to include patients treated with PTCy after the publication of BMT CTN 1703 results (Bolaños-Mead et al. NEJM. 2023). When selecting donors for HCT patients, consideration should be given to patient race/ethnicity.
Clinical and disease characteristics of initial participants at time of enrollment in THRIVE, a prospective, observational cohort study of patients at risk for chronic graft‑versus‑host disease
Xue, Zhenyi , Khera, Nandita , Hamilton, Betty K. , Lorthridge‐Jackson, Shawnta, Bhatt, Valkal, Galvin, John J. , El‐Jawahri, Areej , Lee, Catherine J , Ponce, Doris M , Galvin, John

Blood | December 5, 2025 | Volume: 146 | Issue: Supplement 1 | Pages: 2446-2446

10.1182/blood-2025-2446 Get It

Abstract Background: Chronic graft-versus-host disease (cGVHD) is a leading cause of non-relapse mortality following allogeneic hematopoietic cell transplantation (allo-HCT). Further investigation is needed to understand cGVHD risk, diagnostic features, and outcomes. Patient-reported outcomes (PROs) are important for characterizing the direct and indirect impact of cGVHD on patients, caregivers, and the healthcare system. THRIVE (NCT05919511) is a prospective, longitudinal observational study of patients at risk for cGVHD to describe the clinical course, burden, and practice patterns for cGVHD. THRIVE uses a novel app-based platform designed for rigorous collection of PROs related to symptoms, quality of life, and indirect costs to the healthcare system. We report preliminary patient demographics and clinical characteristics at study enrollment. Methods: THRIVE includes adults aged ≥18 years in the United States who were enrolled 90–180 days after allo-HCT. Follow-up is for 36 months at intervals determined by clinician standard practice; a virtual site is available for patients who return to community sites for follow-up. Presence or absence of GVHD symptoms, cGVHD therapies when applicable, and adverse events are captured in medical records at clinical visits every ~3 months. In all, 13 PROs are assessed (Cutaneous GVHD Questionnaire [CuGQ], General Anxiety Disorder [GAD-7], Lee Total Symptom Scale [TSS; 7-day], modified Frenchay Activities Index [mFAI], modified Patient-Generated Subjective Global Assessment [mPG-SGA], Oral Health Impact Profile [OHIP-14], Ocular Surface Disease Index [OSDI-12], Patient Health Questionnaire [PHQ-9], Patient-Reported Baseline Data [PRBD], Patient-Reported Caregiver Assessment [PRCA], Patient-Reported Economic, Income, and Insurance Data [PREIID], Patient-Reported Outcomes Measurement Information System–Sexual Function [PROMIS-SF], and Pulmonary GVHD Questionnaire [PulmGQ]). Patients are randomized to sequence groups to complete rotating sets of 4–5 PRO measures via digital platform, monthly (Year 1) then every 2 months (Years 2–3). Recruitment is ongoing (target, 1500 patients). Results: This interim report includes the first 340 patients enrolled in THRIVE (data cutoff: June 17, 2025) at 22 sites across the United States since August 1, 2023. Median (range) age was 60.5 (19–83) years (>70 years, n=60 [18%]). Most patients were male (n=190, 56%), White (n=276, 81%), and had college/graduate education (n=219, 64%). Underlying disease was malignant in 328 (96%) patients; most common malignancies requiring allo-HCT were acute myeloid leukemia (n=129, 38%), myelodysplastic syndrome (n=50, 15%), and acute lymphocytic leukemia (n=37, 11%). Most patients (n=212, 62%) were in complete remission at time of allo-HCT, 282 (83%) enrolled after their first allo-HCT, and median (range) time from allo-HCT to enrollment was 111 (77–211) days. Transplant source was most commonly peripheral blood stem cells (n=272, 80%) from unrelated (n=229, 67%) donors, of whom 169 (50%) were male. Overall, 188 (55%) patients had human leukocyte antigen identical HCT, 72 (21%) had mismatched HCT, and 55 (16%) had haploidentical HCT. Conditioning regimens were myeloablative for 177 (34%) patients, reduced intensity for 164 (48%), and non-myeloablative for 16 (5%). At data cutoff, 296 (87%) patients were continuing in THRIVE; 18/44 (41%) patients who discontinued were alive at discontinuation. There were 107 patients (31%) who developed acute GVHD before enrollment (grade I, n=52; grade II, n=35; grade III, n=9; grade IV, n=0; missing/unknown, n=11); 85 (79%) received systemic treatment for acute GVHD. Overall, 28 (8%) patients had cGVHD at enrollment (mild, n=25; moderate/severe, n=3). Organs affected by cGVHD were skin (n=13), eyes (n=4), and gastrointestinal tract (n=3). Conclusions: THRIVE aims to report on clinical practice for managing risk and treatment of cGVHD after allo-HCT in a prospective cohort of ~1500 patients in the United States for ≤3 years. Although real-world assessment of PROs can be associated with challenges including willingness to participate in an observational study and the collection of data without routine clinical visits, the virtual hybrid design is intended to improve patient access and facilitate data collection. In the 22 months since initiation, 340 patients have been enrolled at 22 sites, and the discontinuation rate is low.
Social vulnerability and mortality trends in Acute Myeloid Leukemia in the United States adults (2010 to 2020): The paradoxical role of minority-language communities
Garg, Ayushi , Khera, Nandita , Ballen, Karen K.

Blood | December 4, 2025 | Volume: 146 | Issue: Supplement 1 | Pages: 4621-4621

10.1182/blood-2025-4621 Get It

Abstract Background: Prior work has indicated racial, ethnic and geographic disparities in treatment access and outcomes for acute myeloid leukemia (AML). However, the relationship between social vulnerability and AML outcomes remains understudied. The study examines the association between overall Social Vulnerability Index (SVI) and its domains with crude mortality rate (CMR) in AML patients from 2010 to 2020 and describes temporal trends. Methods: County-level SVI data aggregating 15 census variables into four domains-(1) socioeconomic status, (2) household composition and disability, (3) minority status and language, and (4) housing type and transportation, assign each county a percentile score from 0 (least vulnerable) to 1 (most vulnerable). Counties were categorized into four quartiles based on SVI scores: Q1:0-0.25 (least vulnerable) to Q4:0.75-1 (most vulnerable). CMR per 100,000 patients were abstracted from CDC WONDER database for adult patients (≥25 years) with AML and reported for patients <65 and ≥65 years of age in Q1 and Q4 counties. Counties with <10 deaths (<1% deaths) were excluded. Poisson regression models, using Q1 as reference, assessed the relationship of CMR and overall SVI in each age group, and reported rate ratios (RR) and 95% confidence intervals (CI). Each SVI domain's independent association with CMR was examined in each age group. Temporal trends were assessed by comparing CMR in 2010-2014 and 2016-2020 cohorts. Results: From 2010 to 2020, 103,411 deaths in AML patients occurred (overall CMR: 5.61 per 100,000). Unexpectedly, higher overall SVI was associated with lower mortality: CMR 5.96 in Q1 vs. 5.01 in Q4. As expected, adults aged<65 had a lower mean CMR (1.74 in Q1 vs. 1.77 in Q4) compared to those ≥65 (18.81 in Q1 vs. 15.60 in Q4). In adults aged <65, higher overall social vulnerability was not significantly associated with mortality (Q2 RR 1.01, 95% CI 0.96-1.06, p = 0.75; Q3 RR 1.02, 95% CI 0.98-1.07, p = 0.33; Q4 RR 0.96, 95% CI 0.91-1.00, p=0.054). However, there were significant associations for each SVI domain. A higher SVI in the Minority Status and Language was surprisingly associated with a significantly lower CMR (RR 0.39, 95% CI 0.36-0.43, p<0.001); for the other 3 domains, a higher SVI was associated with higher mortality: Socioeconomic status (RR 1.14, 95% CI 1.04-1.25, p=0.004), Household Composition & Disability (RR 1.31, 95% CI 1.22-1.42, p<0.001) and Housing & Transportation (RR 1.13, 95% CI 1.05-1.22, p=0.001). In adults > 65, higher overall SVI quartiles were linked to lower CMR (Q2 RR 0.96, 95% CI 0.94-0.98, p < .001; Q3 RR 0.91, 95% CI 0.89-0.94, p < .001; Q4 RR 0.84, 95% CI 0.82-0.86, p < .001). As for the domains, Socioeconomic (RR 0.81, 95% CI 0.78-0.85, p<.001) and Minority status and Language (RR 0.77, 95% CI 0.75-0.80, p<.001) were associated with a lower CMR rate. Housing & Transportation was associated with a 12% higher mortality rate (RR 1.12, 95% CI 1.08-1.17, p<.001) and Household Composition & Disability had no significant effect (p=0.214). Mortality decreased over time. In adults aged <65, CMR in Q1 counties improved from 1.94 in 2010-2014 to 1.71 in 2016-2020 and from 1.72 in 2010-2014 to 1.66 in 2016-2020 in Q4 counties. In adults aged ≥65, CMR in Q1 counties improved from 19.62 in the earlier cohort to 18.70 in later cohort but worsened from 16.32 in 2010-2014 to 16.48 in Q4 counties from 2016 to 2020. Conclusions: 1. Mortality for AML has improved over time for most of the population, except among adults in high vulnerability counties. 2. As expected, mortality is higher for older patients. 3. For adults aged <65, a surprising finding was lower mortality in counties with a higher social vulnerability in the minority and language domain, whereas a higher SVI in the socioeconomic, household, and housing domains was associated with higher mortality. 4. For adults aged ≥65, higher SVI in both the socioeconomic and minority domains was associated with a lower mortality rate, and higher SVI in the housing domain with a higher mortality rate. The authors hypothesize that urban areas with a more diverse population, and therefore a higher SVI in Minority Status and Language Domain, may offer more comprehensive care to patients with AML. In addition, the SVI does not capture important clinical and treatment variables that may impact outcomes. Further research is needed to explore more specific, population based indicators to identify at-risk patients with AML.
Upstream integration of implementation planning in clinical trials through the blood and marrow transplant clinical trials network
DeSalvo, Anna M. , Ragon, Brittany Knick , Hamilton, Betty K. , Borogovac, Azra , Horowitz, Mary, Khera, Nandita , Keyzner, Alla , Efebera, Yvonne A. , Horwitz, Mitchell E. , Wall, Sarah, Stewart, Valerie , Heuer, Mykala, Bhatt, Neel S. , Juckett, Mark , Devine, Steven M. , Dandoy, Christopher E. , Plastaras, John Peter , Wall, Sarah , Heuer, Mykala, Molfenter, Todd

Blood | December 4, 2025 | Volume: 146 | Issue: Supplement 1 | Pages: 7913-7913

10.1182/blood-2025-7913 Get It

Abstract Background: Dissemination and implementation science (D+I) efforts often begin after clinical trial (CT) efficacy data are available. However, delaying this work limits opportunities to identify barriers and facilitators that can enhance adoption of evidence-based practices. Integrating D+I during trial development, activation, and conduct supports more timely, effective, and scalable implementation. The Blood and Marrow Transplant Clinical Trials Network (BMT CTN) includes >125 centers, conducting transplant and cellular therapy (TCT) trials with practice-changing potential, with >17,000 patients enrolled since 2001. Data from Center for International Blood and Marrow Transplant Research (CIBMTR) provides a way to measure real-world adoption. With this comprehensive structure in place, the BMT CTN launched a D+I Committee that established a D+I process to better understand uptake of CT findings and to leverage that information to promote adoption. Methods: Informed by established D+I frameworks, a structured, cyclical process to integrate implementation considerations spanning the CT lifecycle was developed. A multidisciplinary team including experts in D+I, clinicians, and investigators iteratively refined the proposed D+I algorithm. The process begins upstream when a trial concept is approved to move forward, it is shared with the D+I Committee for initial assessment. This includes a landscape review for studies in development using CIBMTR data, literature, and expert experience. The Committee then reviews the developing protocol with the BMT CTN protocol team to proactively identify implementation barriers and facilitators to implementation in real-world settings and outline plans for dissemination. The Committee also provides recommendations for integrating relevant implementation data collection and analyses within the protocol. After study completion, both clinical efficacy and implementation outcomes are analyzed, followed by active dissemination. Additional assessment using surveys, qualitative methods, and CIBMTR data occurs after dissemination to understand adoption. Insights gathered also inform further refinement of the process. Tools and templates are in development to build capacity and ensure consistency for Committee members. Results: To date, the Committee has published three peer-reviewed articles addressing implementation implications of three BMT CTN trials (PMC7735536, PMC8578257, PMC9364468) and a fourth is in press. Using the designed D+I process flow, a current effort used a survey sent to US CIBMTR centers, to evaluate factors influencing the implementation of results of the BMT CTN 1703 trial of post-transplant cyclophosphamide as graft-versus-host disease (GvHD) prophylaxis and assessed dissemination and adoption considerations for the upcoming BMT CTN 2203 CT of ruxolitinib as GvHD prophylaxis. CIBMTR data is being analyzed to demonstrate practice patterns before, during, and after BMT CTN 1703 study completion. This effort has allowed for further refinement of the BMT CTN D+I process flow and has highlighted critical opportunities for incorporating dissemination and implementation objectives and endpoints into BMT CTN protocols. We also used the process to guide a review of severe aplastic anemia treatment gaps and ongoing studies. This review underscored how proactive transplant and non-transplant stakeholder engagement and early implementation planning, core elements of this process, can bridge evidence to practice and advance access to care. A complementary survey on current practice patterns in severe aplastic anemia is ongoing. Conclusions: With the existing structure of the BMT CTN and associated practice registry of the CIBMTR, the BMT CTN D+I Committee developed a systematic process to build D+I considerations into trial workflows to increase real-world impact. This process aims to ensure that barriers to uptake of trial results can be identified and addressed appropriately, improving care quality for patients undergoing TCT.
Safety and efficacy of post transplant cyclophosphamide vs tacrolimus plus methotrexate for gvhd prophylaxis in myelofibrosis
Ilyas, Usman , Mead‐Harvey, Carolyn , Vanegas, Yenny Moreno , Humayun, Muhammad Abdullah, Chandani, Kanishka Uttam, Quintanilla, Diana Zamora, Ayala, Ernesto, Foran, James M. , Kharfan‐Dabaja, Mohamed A. , Khera, Nandita , Leis, José F., Punwani, Nathan , Sproat, Lisa, Palmer, Jeanne , Chhabra, Saurabh , Hilal, Talal , Iqbal, Madiha , Murthy, Hemant S. , Roy, Vivek

Blood | December 4, 2025 | Volume: 146 | Issue: Supplement 1 | Pages: 6016-6016

10.1182/blood-2025-6016 Get It

Abstract Background In patients with myelofibrosis (MF) undergoing allogeneic hematopoietic cell transplantation (allo-HCT), graft-versus-host disease (GVHD) prophylaxis has traditionally included a calcineurin inhibitor (CNI) plus methotrexate (MTX). We conducted a retrospective study comparing the safety and efficacy of post-transplant cyclophosphamide (PTCy)-based GVHD prophylaxis to CNI-based regimens. Methods This study was approved by the Mayo Clinic IRB. Patients with myelofibrosis who underwent allo-HCT after 2016 at Mayo Clinic in Arizona and Florida were included. Categorical variables were compared using Chi-square or Fisher's exact tests. Overall survival (OS) was defined from transplant to death, and progression-free survival (PFS) from transplant to relapse or death. OS and PFS were estimated using Kaplan-Meier methods; univariate differences were assessed with log-rank tests. The cumulative incidence of acute and chronic GVHD and time to neutrophil and platelet engraftment were analyzed using competing risk models, with death as a competing event; differences were tested using Gray's test. Cox proportional hazards models were used to estimate adjusted hazard ratios (HRs). Results A total of 110 patients were included. 53 patients received PTCy-based prophylaxis (43 PTCy/Tac/MMF, 1 PTCy/CSA/MMF, 9 PTCy/Siro/MMF), while 57 received non-PTCy regimens (54 Tac/MTX +/- 39 T-cell depletion, 2 CSA/MMF, 1 Tac/MMF). Median age at transplant was 63 (range: PTCy 40-76; others 38-74) years in both groups; 59 were male (58.5% PTCy vs 49.1% others). Median Karnofsky Performance Status (KPS) and HCT–Comorbidity Index (HCT-CI) in patients receiving PTCy vs non-PTCy were 90 vs 80 and 1 vs 3, respectively. Conditioning intensity was similar: 22.6% myeloablative vs 21.7%, and 77.4% reduced-intensity vs 76.7% in PTCy and non-PTCy, respectively (p=1.000). Stem cell source was peripheral blood in all patients except one in the non-PTCy group who received bone marrow. 58 patients had primary MF (54.7% PTCy vs 50.9% others), and 52 had secondary MF (45.3% PTCy vs 49.1% others), including essential thrombocythemia (28.3% PTCy vs 31.6% others), polycythemia vera (17.0% vs 15.8%), and MPN-NOS (0% vs 1.8%). Median follow-up was 26.2 months (95% CI: 20.7-52.7) in the PTCy and 60.6 months (95% CI: 53.1-70.6) in the non-PTCy cohort. CD3 and CD33 donor chimerism ≥95% at day +100 was 66.7% and 86.7% in PTCy vs 63.6% and 92.7% in others, respectively (p=0.50); day +100 driver molecular mutations were present in 32.1% of PTCy and 32.0% of others (p>0.99). Rates of acute GVHD grades ≥2 and ≥3 were not statistically significant, in the PTCy group (37.7% vs 49.1% p=0.229 and 17.0% vs 22.8% p=0.445, respectively). Chronic GVHD (any grade) occurred in 42.3% of PTCy vs 38.2% of others (p=0.664), while moderate-to-severe cGVHD was lower with PTCy (21.2% vs 27.3% p=0.461). Platelet engraftment was significantly delayed in the PTCy group (median 33 vs 21 days, p=0.030). Median time to neutrophil engraftment was also delayed in the PTCy group (20 vs 17 days, adjusted p=0.025). Age-adjusted HR for time to platelet engraftment was 0.60 (95% CI: 0.41–0.90; p=0.014), and for neutrophil engraftment 0.65 (95% CI: 0.44–0.95; p=0.025), indicating significantly delayed hematopoietic recovery with PTCy. Graft failure occurred in 9.4% vs 5.3% (p = 0.48); primary 7.5% vs 3.5%, secondary 1.9% vs 1.8% in PTCy vs non-PTCy groups, respectively. Relapse or progression occurred in 11.3% of PTCy vs 17.5% of others (p=0.355); four patients in the non-PTCy group progressed to AML post-transplant, versus none in the PTCy group (p=0.119). 1-year TRM was 22.6% in the PTCy group and 14.0% in the non-PTCy group, p = 0.324. Median OS was 72.8 months in the PTCy group (95% CI: 49.9 months, upper limit not estimable [NE]) and 93.4 months in the non-PTCy group (95% CI: 48.5 months, upper limit NE), although the difference was not statistically significant (p=0.739). After adjusting for age and HCT-CI, there were no significant differences in PFS (adjusted HR 1.37; 95% CI: 0.73–2.56; p=0.331) or OS (adjusted HR 1.27; 95% CI: 0.64–2.51; p=0.498). Conclusions In myelofibrosis patients undergoing allo-HCT, PTCy-based GVHD prophylaxis showed similar rates of grade ≥2 acute and moderate-severe chronic GVHD compared to non-PTCy regimens. Engraftment was significantly delayed, while OS and PFS were comparable. PTCy appears to be a safe and viable alternative to CNI-based prophylaxis.
Central Venous Catheter Removal After Hematopoietic Stem Cell Transplant: A Review and Perspective
Cook, Eleanor , Rotz, Seth J. , Blacken, Robyn, Johnson, Darren, Alonso, Priscila Badia , French, Karis, Kent, Georgia , Huber, John , Hammoud, Miza Salim , Parker, Loretta , Kusnier, Katilyn , Lehmann, Leslie , Kapadia, Malika, Khera, Nandita , Nishitani, Miki , Craddock, John , Juckett, Mark, Maier, Steffani, O'Mathúna, Dónal, Phelan, Rachel , Young, Kylan, Jodele, Sonata , Snyder, Alana J. , Sampson, Megan , Dandoy, Christopher E. , Fitch, Taylor Jennelle , Badia, Priscila , Huber, John , Hammoud, Miza Salim , Myers, Kasiani , Craddock, John A , Jodele, Sonata , Schoettler, Michelle L , Sampson, Megan , Dandoy, Christopher E , Auletta, Jeffery J. , Bidgoli, Alan , French, Karis, Hartley, David , Maier, Steffani T. , O'Mathúna, Dónal, Nelson, Adam S. , Phelan, Rachel , Young, Kylan

Pediatric Blood & Cancer | November 24, 2025 | Volume: 73 | Issue: 3 | Pages: No page available

10.1002/pbc.70001 Get It

ABSTRACT Central venous catheters (CVCs) are a cornerstone of hematopoietic stem cell transplantation (HSCT), facilitating the delivery of chemotherapy, parenteral nutrition, medications, and blood products. Despite CVCs’ essential role, prolonged use can lead to complications such as increased risk of infection, thrombosis, mechanical issues, and psychosocial burden. However, the timing of CVC removal remains highly variable across institutions. Additionally, there are no standardized guidelines to balance the benefits of extended access against the risks of prolonged use. This narrative review examines the existing literature on CVC management post‐HSCT, highlighting key factors influencing the removal timing, such as patient characteristics, disease‐specific considerations, and institutional practices. The review emphasizes the urgent need for multi‐institutional research and standardized protocols to improve outcomes, reduce complications, and enhance the quality of care for HSCT patients.
Perceptions of barriers and facilitators of video telemedicine visits among cancer patients since onset of the COVID-19 pandemic: A qualitative study.
Dholakia, Ruchita, Decker, Paul A. , Khera, Nandita , Tilburt, Jon C. , Haddad, Tufia C. , Pritchett, Joshua C. , Kamath, Celia , Brockman, Tabetha A. , Huang, Ming , Moriarty, James P. , Decker, Paul A. , Brewer, LaPrincess C. , Borah, Bijan J. , Patten, Christi A. , Sharma, Pravesh

JCO Oncology Practice | October 7, 2025 | Volume: 21 | Issue: 10_suppl | Pages: 566-566

10.1200/op.2025.21.10_suppl.566 Get It

566 Background: Video telemedicine visits (VTVs) have the potential to overcome disparities in access to cancer care for many socially, geographically, and economically disadvantaged populations. Internet connectivity, digital literacy, and socioeconomic factors are traditionally cited as influencing VTV utilization. However, digital diffusion has occurred rapidly across several sectors, including healthcare, since the onset of the COVID-19 pandemic, and less is known about how these factors have evolved to influence VTV utilization behavior over this time. Through a qualitative approach, we aimed to: (1) explore the lived experiences of cancer patients who transitioned to VTV use as compared to those who persistently did not, and (2) identify similarities and differences between these two groups that could inform targeted interventions to support access and patient acceptance of VTVs in the setting of cancer care. Methods: During June 2024, we conducted semi-structured interviews with 24 patients (Mean age 71.3 years, range 42-88; female 50%) receiving ongoing, longitudinal cancer care at our academic medical center since July 2021 who had not used VTVs through at least October 2022. Using purposeful sampling, patients were equally divided according to VTV utilization behaviors defined as “transitioned users” (TU; i.e. those who transitioned to VTV utilization after October 2022) and “persistent non-users” (PNU). Transcripts were analyzed using a combined deductive–inductive thematic approach and categorized into themes of barriers and facilitators across three domains consistent with the COM-B model: capabilities (C), opportunities (O), and motivations (M). Results: TU patients frequently cited a reduction in travel and logistical considerations as a primary reason for uptake of VTVs. PNU patients frequently reported digital literacy barriers, particularly among older patients, as primary reasons for lack of VTV utilization. TU patients reported more experience and comfort in technical skills as compared to PNU participants. PNU patients also reported less frequent offering of VTV as an option by their care teams over time, implying they may have transitioned to VTV utilization if it had been offered with more regularity. Both study groups desired improved internet access and technical support to enhance (or, in the case of PNU patients, enable) VTV utilization. Conclusions: These findings suggest that multi-modal care delivery may best serve current patient populations, not requiring, yet offering VTV services as an adjunct to in-person care. Longer term, individual barriers to VTV use will also need to be addressed to further optimize access for patients who desire to incorporate this care modality.
Impact of Publicly Reported Outcomes on Patient Selection for Hematopoietic Cell Transplantation
Strouse, Christopher , Logan, Brent R. , Estrada‐Merly, Noel, Peterson, Andrew C. , Preussler, Jaime M. , Truong, Tony H. , Khera, Nandita , Wood, William A. , Rangarajan, Hemalatha G. , Akard, Luke P., Sharma, Akshay , Rizzo, J. Douglas , Saber, Wael , Logan, Brent , Estrada-Merly, Noel , Preussler, Jaime M , Truong, Tony H , Khera, Nandita , Wood, William A , Bhatt, Neel S , Sharma, Akshay , Rizzo, J Douglas , Saber, Wael , Strouse, Christopher S , Juckett, Mark , Peterson, Andrew, Troy, Jesse D , Bhatt, Neel S.

JCO Oncology Practice | October 2, 2025 | Volume: 22 | Issue: 5 | Pages: 844-851

10.1200/op-25-00115 Get It

PURPOSE Public reporting of health care outcomes can have unintended effects such as inappropriate risk aversion in patient selection. METHODS The center-specific survival analysis annually assigns all hematopoietic cell transplantation (HCT) centers in the United States a +1, −1, or 0 score for observed outcomes that are above, below, or within a center-specific predicted range of outcome. For each index year (2012-2016), centers receiving a −1 score after 0 scores in the preceding 2 years were compared with contemporaneous centers with as-predicted outcomes (0 score). Changes in the patient population characteristics in the 3 years before versus the 3 years after the index years were compared between the newly below-expected centers (NBCs) and the controls. A multivariate model adjusted for baseline patient population characteristics and center volume. RESULTS No differences in patient selection behavior were identified when comparing the NBCs with the controls across eight key patient population characteristics. For the statistically modeled (predicted) 1-year overall survival (OS), reflecting a holistic measure of centers' patient population risk, we observed no statistically significant difference in change (−0.23% [95% CI, −1.4 to 0.9]; P = .70). The observed OS increased in both NBCs and controls by 0.9% and 4.5%, respectively, without statistically significant difference in change. CONCLUSION Centers receiving a −1 score were not observed to deviate significantly from patient selection trends in the HCT field. These findings suggest that public reporting of HCT outcomes in the United States does not result in unintended bias against HCT for high-risk patients.
Peer Support for Caregivers: An Acute Care Quality Improvement Initiative
Smiley, Adrianne, Vitagliano, Luis, Wold, Angela K., Mailloux, Tish, Hansen, Beverly, Strader, Kelli D., Khera, Nandita , Burns, Michelle R., Vitagliano, Lynne M, Hansen, Beverly B, Smiley, Adrianne , Griffin, Joan M. , Strader, Kelli D. , Chesak, Sherry S.

American Journal of Medical Quality | July 1, 2025 | Volume: 40 | Issue: 4 | Pages: 208-209

10.1097/jmq.0000000000000249 Get It