Citations

Mayo Clinic

Clear

7719 citation(s) found for "Ma, Daniel M".

Allogeneic haematopoietic cell transplantation in advanced systemic mastocytosis in the new era: A CIBMTR study
Üstün, Celalettin , Zhang, Mei‐Jie , Peterson, Andrew C. , Baek, Alexander, Coltoff, Alex, Gandhi, Arpita , Ho, Vincent T., Lin, Adam, Gowda, Lohith , Mensah, Felix, Nadiminti, Kalyan , Trunk, Andrew D. , Uberti, Joseph P., Marcucci, Guido , Akın, Cem , Pullarkat, Vinod , Juckett, Mark, Saber, Wael , Burns, Linda J. , Alkhateeb, Hassan B , Borthakur, Gautam , Nadiminti, Kalyan V , Nishihori, Taiga , Trunk, Andrew , Hamadani, Mehdi , Oran, Betul , Peterson, Andrew, Agha, Mounzer , Alkhateeb, Hassan B. , Chhabra, Saurabh , Coltoff, Alex, Lima, Marcos de , Kassim, Adetola A , Lin, Adam, Borthakur, Gautam , DeAngelo, Daniel J. , McGuirk, Joseph P. , Nishihori, Taiga , Pantin, Jeremy , Gotlib, Jason , Akin, Cem , Hamadani, Mehdi , Valent, Peter , Grunwald, Michael R. , Oran, Betül

British Journal of Haematology | September 23, 2025 | Volume: 207 | Issue: 6 | Pages: 2486-2495

10.1111/bjh.70154 | PMID 40983528 Get It

Summary We investigated the outcomes of patients with advanced systemic mastocytosis (AdvSM) undergoing allogeneic haematopoietic cell therapy (alloHCT) in the US. Twenty‐seven adult (median age, 58 years) patients with AdvSM received alloHCT from 2014 to 2021. Most patients were white (93%), male (63%) and had systemic mastocytosis (SM) with associated haematological neoplasm (SM‐AHN, 70%), received peripheral blood grafts (89%) and non‐myeloablative regimens. KIT mutation was detected in 14 of the reported 16 patients (87.5%). Post‐transplantation cyclophosphamide (PTCy) was used in approximately 50% of the cohort. Approximately 1 year after alloHCT, median bone marrow mast cell burden decreased from 15% pre‐alloHCT to 1.5% (range 0–8) at 1 year post‐alloHCT. The median serum tryptase decreased from 55 ng/mL (range 3–400 ng/mL) pre‐alloHCT to 18 ng/mL (range 0–481 ng/mL) at 1 year post‐alloHCT. At 1 year, progression‐free survival (PFS) and overall survival (OS) were 59% (95% confidence interval [CI], 41%–77%) and 74% (95% CI, 56%–89%) respectively. In the subset of patients with SM‐AHN, 1‐year PFS and OS were 74% (52%–91%) and 79% (95% CI, 58%–94%) respectively. The cumulative incidence of relapse/progression for the entire population of patients with SM ( n = 27) was 4% (95% CI, 0–14) at 100 days and 15% (95% CI, 4–31) at 1 year. For the subset of patients with AHN, the cumulative incidence was 11% (95% CI, 1–28) at 100 days and 21% (95% CI, 6–42) at 1 year. Relapse/progression was the cause of death in six of 12 patients (5 died of AHN and 1 died of SM progression). Eighteen and nine patients received a KIT inhibitor before alloHCT and after alloHCT respectively. In addition, six patients received a KIT inhibitor for the treatment of relapse/progression of SM after alloHCT. Although it is difficult to delineate the exact effect of alloHCT and KIT inhibitors because of the size of the study in this era, the use of alloHCT and KIT inhibitors pre‐ and post‐ alloHCT is increasing. In the near future, larger studies are required to provide further insights into this subject.
Incidence of Complications, Recurrent Instability, and All-Cause Reoperations After Tibial Tubercle Osteotomy for Patellofemoral Instability: A Systematic Review and Meta-analysis of the Literature
DeNovio, Anthony , Holle, Alejandro M. , Saris, Daniël B.F. , Krych, Aaron J. , Hevesi, Mario , Okoroha, Kelechi R. , Tagliero, Adam J. , DeNovio, Anthony C, Holle, Alejandro , Krych, Aaron J , Hevesi, Mario , Okoroha, Kelechi, Tagliero, Adam , Pan, Xuankang

Orthopaedic Journal of Sports Medicine | September 22, 2025 | Volume: 13 | Issue: 9 | Pages: No page available

10.1177/23259671251374307 | PMID 40995414 Get It

Background: Patellofemoral instability (PFI) is a condition that leads to recurrent dislocation events and is typically secondary to extensor mechanism malalignment and rotational deformities. To correct malalignment, a tibial tubercle osteotomy (TTO) can be performed; however, an understanding of the associated complications, recurrence rates, and potential for reoperation is necessary. Purpose: To systematically review the literature to determine the incidence of postoperative complications, rate of recurrent instability, and all-cause reoperation rate after TTO for PFI. Study Design: Systematic review; Level of evidence 4. Methods: The Cochrane Database of Systematic Reviews, PubMed, Embase, MEDLINE, and CINAHL were queried for studies that reported complications after TTO. No date limits were applied. A total of 1050 studies were identified. Complications were categorized into “minor” and “major” groups using a modified Clavien-Dindo Classification. A meta-analysis was conducted to examine complication rates, recurrent instability, and all-cause reoperation, utilizing a DerSimonian and Laird random-effects model. A meta-analysis for recurrent instability was subgrouped according to follow-up periods of ≤2 years and >2 years. Results: A total of 38 studies were included, comprising 1546 patients, representing 1733 knees. The study dates ranged from 1989 to 2024, and the level of evidence ranged from 2 to 4. The weighted mean age at surgery was 24.1 years (range, 20-54 years), and 67% of patients were women. The weighted mean follow-up of all studies was 4.2 years. Overall, 197 minor complications, 39 major complications, 90 recurrent instability events, and 275 reoperations were identified. The minor complication rate was 9% (range, 0%-65% [95% CI, 6%-13%]). The major complication rate was 3% (range, 0%-10% [95% CI, 3%-4%]). Seven studies had a ≤2-year follow-up, and the remaining 31 studies had a >2-year follow-up. The recurrent instability rate was 3% in studies with ≤2 years of follow-ups (range, 0%-26% [95% CI, 1%-13%]). In studies with >2 years of follow-up, the recurrent instability rate was 8% (range, 0%-31% [95% CI, 6%-12%]). The all-cause reoperation rate was 16% (range, 0%-54% [95% CI, 11%-21%]). Conclusion: This systematic review and meta-analysis identified the incidence of complications (17%), the rate of recurrent instability (3%-8%), and the all-cause reoperation rate (16%) after TTO. TTO, performed alone or in combination with medial patellofemoral ligament reconstruction for the treatment of PFI, reliably improved clinical outcome scores in the setting of variable complication rates. Interestingly, rates of recurrent instability increased over time, and most reoperations were undertaken for hardware removal rather than revision patellar stabilization surgery.
First-line atezolizumab/bevacizumab or durvalumab/tremelimumab in advanced hepatocellular carcinoma: a real world, multicenter retrospective study
Kournoutas, Ioannis , Marell, Paulina S. , Peersen, Anina, Shah, Priyanshi , VanDommelen, Kyle, Gupta, Garima , Akce, Mehmet , Yang, Ju Dong , Chen, Pin‐Jung , Naleid, Nikolas , Peterson, Nicole , Sahai, Vaibhav , Halfdanarson, Thorvardur, Kankeu, Lionel Fonkoua, Washburn, Leslie, Conboy, Caitlin B. , Goenka, Ajit H. , Thompson, Scott A. , Ahn, Daniel H. , Jatoi, Aminah , Marell, Paulina, Gile, Jennifer , Yang, Ju Dong , Sahai, Vaibhav , Jin, Zhaohui , Fonkoua, Lionel Aurelien A Kankeu , Washburn, Leslie A, Goenka, Ajit, Starlinger, Patrick , Roberts, Lewis, Jatoi, Aminah , Ou, Fang-Shu , Tran, Nguyen , Conboy, Caitlin B , Kamath, Suneel Deepak , Mahipal, Amit , Wee, Wen , Jin, Zhaohui , Fonkoua, Lionel Aurelien Kankeu , Torbenson, Michael , Thompson, Scott, Venkatesh, Sudhakar K. , Starlinger, Patrick , Roberts, Lewis R. , Gores, Gregory J. , Babiker, Hani M. , Borad, Mitesh J. , Bekaii‐Saab, Tanios , McWilliams, Robert R. , Ou, Fang‐Shu

The Oncologist | September 20, 2025 | Volume: 30 | Issue: 11 | Pages: No page available

10.1093/oncolo/oyaf286 | PMID 40973475 Get It

Abstract Background Unresectable hepatocellular carcinoma (uHCC) is a leading cause of cancer death. FDA-approved first-line systemic therapies include atezolizumab/bevacizumab (atezo/bev) and durvalumab/tremelimumab (durva/treme); however, there is a lack of comparative data. Methods We reviewed outcomes of patients with uHCC who initiated atezo/bev or durva/treme between 2017 and 2024, across six institutions. Overall survival (OS) and time to treatment discontinuation (TTD) were analyzed using the Kaplan–Meier and Cox models, adjusting for baseline characteristics. Results Four hundred fifty-two uHCC pts were included. Median age: 68 years; 77% male; 81% white. Most common etiologies were viral hepatitis (38.9%) and metabolic dysfunction-associated steatohepatitis (19.5%). Disease progression was the primary reason for treatment discontinuation, atezo/bev (56%) and durva/treme (42%). Outcomes were not statistically significant (median OS [month, m]: 14.0 vs 14.6 [P = .66]; median TTD [m]: 4.9 vs 3.9 [P = .42] for atezo/bev vs durva/treme). Outcomes were significantly different between Child-Pugh classes (CP: A, B7, B8/9, C) respectively, median OS(m): 19.0, 6.1, 5.1, 2.0 (P < .001); median TTD(m): 6.1, 2.3, 3.0, 1.3 (P < .001). Conclusions In this real-world study of uHCC, no significant difference in clinical outcomes was observed between atezo/bev and durva/treme in the first-line setting. CP scores were a key prognostic variable with both regimens.
Partial Oral Therapy for Infective Endocarditis Among Adult Infectious Diseases Physicians in the United States: An Emerging Infections Network Survey
McHugh, Jack , Baddour, Larry M. , Chesdachai, Supavit , Polgreen, Philip M. , Wilson, Walter R., DeSimone, Daniel C. , McHugh, Jack , Polgreen, Philip M , DeSimone, Daniel C , Baddour, Larry , Chesdachai, Supavit , Beekmann, Susan E.

Open Forum Infectious Diseases | September 19, 2025 | Volume: 12 | Issue: 9 | Pages: No page available

10.1093/ofid/ofaf580 | PMID 41001618 Get It

Abstract Background Recent clinical trial evidence supports broader use of partial oral therapy (POT) for infective endocarditis (IE), yet real-world uptake in the U.S. has not been investigated. Methods Adult infectious diseases (ID) physician members of the Infectious Diseases Society of America Emerging Infections Network were surveyed in April–May 2025. A 10-item instrument captured frequency of POT, organism-specific influence, decision factors, barriers, and facilitators. Results Among 1531 members, 516 (34%) responded; 452 (88%) of them managed IE. POT was uncommon: 16% never used, 53% used in ≤10% of cases, and only 10% used in >25% of patients. Frequent POT rose with caseload (23% in physicians treating >50 IE cases year vs ≤9% in lower-volume groups, P < .001) and with fewer years in clinical practice (13% in <5 yrs vs 5% in ≥25 yrs, P = .013). Comfort with POT depended on the pathogen: 66% were comfortable switching for Streptococcus spp., 52% for Gram-negative bacilli, 19% for methicillin-resistant Staphylococcus aureus. Three quarters of those who used POT finished with a single agent. In people who inject drugs, 34% of physicians often or always considered an oral regimen. Availability of an active oral agent (75%) and the pathogen involved (69%) were the leading decision drivers; principal barriers were fear of relapse (72%), adherence concerns (53%), and insufficient evidence (48%). Respondents most desired clearer guidelines (75%) and additional data (71%). Conclusions U.S. adult ID physicians adopt POT for IE sparingly. Updated IE treatment guidelines, additional clinical trial data, and broader access to complex outpatient antimicrobial therapy services may facilitate wider adoption.
Extended Trochanteric Osteotomy Combined with Medial Reduction Corticotomy to Correct Femoral Deformity at the Time of Revision Total Hip Arthroplasty
Restrepo, Diego J. , Perez, Sergio F. Guarin , Abdel, Matthew P. , Berry, Daniel J. , Sierra, Rafael J. , Guarin Perez, Sergio F , Abdel, Matthew P , Berry, Daniel J , Sierra, Rafael J , Restrepo, Diego J. , Tai, Ta‐Wei

JBJS Open Access | September 19, 2025 | Volume: 10 | Issue: 3 | Pages: No page available

10.2106/jbjs.oa.25.00193 | PMID 40978000 Get It

Background: An extended trochanteric osteotomy (ETO) is commonly used to improve exposure and facilitate femoral component removal in revision total hip arthroplasty (THA). An additional medial corticotomy may be used in conjunction with an ETO to correct a femoral deformity, particularly varus remodeling in association with a failed femoral component. This study evaluated the outcomes of combining an ETO with a medial corticotomy in revision THA, with emphasis on implant fixation, femoral alignment, bone union, and clinical outcomes. Methods: Of the 612 ETOs performed between 2003 and 2013, 13 patients (9 men and 4 women) underwent ETO combined with a medial corticotomy to correct varus remodeling, representing 2% of all ETOs during that period. The average follow-up was 8 ± 3.5 years. The mean age at surgery was 67 ± 13.5 years. The mean body mass index was 32 ± 7.7 kg/m 2 . Radiographs were reviewed to measure preoperative and postoperative femoral deformity, time to consolidation, and femoral fixation. Clinical outcomes were evaluated using the Harris Hip Score (HHS). Results: All patients had preoperative varus femoral deformity (mean 16.3° ± 5.7°). The mean postoperative alignment was 4.3 ± 1.6° varus achieving an average correction of 12° (95% CI −15.7 to −8.3, p < 0.0001). Both the ETO and the medial corticotomy consolidated in 10 of 11 patients (91%) with available 1-year radiographs at a mean of 11 ± 7.7 months. The mean HHS improved significantly from 42 preoperatively to 82 at 5-year follow-up (p = 0.0002). Complications related to the ETO and corticotomy occurred in 4 patients (30%), including 1 intraoperative fracture, 1 postoperative greater trochanteric fracture, 1 nonunion of the medial corticotomy, and 1 postoperative wound-hematoma. All femoral components remained well fixed at final follow-up. Conclusion: The combination of ETO and medial corticotomy in revision THA effectively corrected femoral alignment in patients with a preoperative varus deformity and was associated with significant functional improvement at the final follow-up. Level of Evidence: Level IV . See Instructions for Authors for a complete description of levels of evidence.
Abstract C138: Influence of demographic factors and knowledge on prostate cancer screening attitude and research participation among Black men
Bolajoko, Opeyemi , Wimberly, Quincy A., Lee, Daniel W. , Mahin, Collisa, Fudge, Michelle, Flynn, Shaina, Merriweather, Arnold, Hew, Trevanne Rose Matthews, Albertie, Monica, Greene, Dery, Ford, W. Scott, Moultrie, Toshiko, Willis, Floyd B. , Fudge, Michelle , Greene, Dery, Ford, Wayne

Cancer Epidemiology, Biomarkers & Prevention | September 18, 2025 | Volume: 34 | Issue: 9_Supplement | Pages: C138-C138

10.1158/1538-7755.disp25-c138 Get It

Abstract Introduction: The disproportionate prostate cancer (CaP) burden, which includes high mortality rates, is significantly attributed to the characteristics of the social determinants of the health status of the Black Male population. Designing targeted interventions to address poor health outcomes through a collaborative population-based approach could reduce CaP health disparities. The Community Living Lab (CoLLab) Learning Health System was developed to address ethnically sensitive, targeted approaches that will eliminate CaP health disparities among Black Men. This study assessed demographic factors and Knowledge on prostate cancer screening attitudes and research participation among black men. Method: The collab project employed a social ecological model to design intervention to address barriers to CaP screening and participation in clinical trials. A validated, pretested questionnaire was used to collect information on demographics, CaP knowledge, attitudes, testing, and a history of participation in research and clinical trials. Baseline data were collected from 3 American Legion posts (ALPs). The association between variables was evaluated using the Chi-square test. Results: Participants between 60-69 years were 28.38%, and those aged 40-49 were 12.16%. Most are US-born Black (89.8%), with others identifying as African-born Black (6.76%) and Caribbean-born Black (1.35%). Approximately 43.24% were retired, and 36.49% were working full-time. CaP knowledge was high in 43.9%, moderate in 46.6%, and low in 9.5%. Only 45% of the participants have undergone Prostate-Specific Antigen test, and 24.6% have had a Digital Rectal Examination. Reported past clinical trials and research participation were very low (9.38%). Participants with higher knowledge levels had higher information scores (p &lt; .0001), favored various research settings (p = 0.0008), had favorable attitudes towards CaP testing (p &lt; .0001), and were more likely to participate in research (p = 0.0117). Work status was found to impact CaP Knowledge (p = 0.0117). Participants with stronger beliefs about prostate cancer may have different attitudes toward testing (p = 0.0444). Age did not impact attitudes toward CaP testing (p = 0.4298). Conclusion: The knowledge of CaP was found to impact the attitudes, testing, and disposition of the participants towards research. The baseline data generated from the clinical study will curate insightful evidence that will create innovative approaches to prostate cancer education among males in Black communities. Citation Format: Opeyemi O. Bolajoko, Quincy A. Wimberly, Daniel W. Lee, Collisa Mahin, Michelle Fudge, Shaina E. Flynn, Arnold J. Merriweather, Trevanne R. Matthews Hew, Monica L. Albertie, Dery Greene, Wayne Ford, Toshiko Moultrie, Floyd B. Willis, Folakemi T. Odedina. Influence of demographic factors and knowledge on prostate cancer screening attitude and research participation among Black men [abstract]. In: Proceedings of the 18th AACR Conference on the Science of Cancer Health Disparities; 2025 Sep 18-21; Baltimore, MD. Philadelphia (PA): AACR; Cancer Epidemiol Biomarkers Prev 2025;34(9 Suppl):Abstract nr C138.
Evolution of Potentially Actionable Genomic Alterations in Advanced Prostate Cancer: A Real-World Analysis of Serial Circulating Tumor DNA Testing
Muniz, Miguel, Orme, Jacob J. , Wei, Nancy , Koch, Regina M. , Scharf, Zachary, Gupta, Sounak , Kase, Adam M. , Pessoa, Rodrigo Rodrigues, Kwon, Eugene D., Andrews, Jack R. , Muniz, Miguel , Orme, Jacob J , Basourakos, Spyridon P. , Wei, Nancy , Scharf, Zachary , Gupta, Sounak , Kase, Adam M , Childs, Daniel S , Tsai, Jill , Bucheit, Leslie , Riaz, Irbaz Bin , Childs, Daniel S.

Cancers | September 18, 2025 | Volume: 17 | Issue: 18 | Pages: 3048

10.3390/cancers17183048 | PMID 41008889 Get It

Background: Longitudinal genomic profiling through serial circulating tumor DNA (ctDNA) testing offers a noninvasive method to monitor clonal evolution in advanced prostate cancer. This study evaluated the frequency and nature of newly emergent and potentially actionable genomic alterations detected through serial testing in a real-world setting. Methods: We conducted a retrospective analysis of advanced prostate cancer patients who underwent multiple Guardant360 ctDNA tests between October 2020 and March 2023. The study focused on identifying new genomic alterations absent in the initial test, with particular attention to alterations relevant for on-label therapies, therapies approved in other oncologic indications (i.e., off-label), or a clinical trial. Results: Among 479 patients with at least two ctDNA tests, the median interval between the first and second evaluable tests was 207 days. New and potentially actionable alterations emerged in 57.8% of patients, including potential targets for on-label therapies (16.7%), off-label therapies (16.5%), and clinical trials (55.7%). Tumor mutational burden (TMB) increased from “low” to “high” in 11% of patients, although none had microsatellite instability or mismatch repair deficiency. In a Mayo Clinic subset, ten patients received olaparib based on treatment-emergent alterations, but none achieved a prostate-specific antigen (PSA) response. Two patients who transitioned from low to high TMB received pembrolizumab, both with progressive disease as best response. Conclusions: In a large real-world cohort, serial ctDNA testing frequently identified new alterations that were not detected at baseline and are potentially actionable therapeutic targets, highlighting the value of serial genomic profiling for capturing clonal dynamics. Additional research is needed to better establish a framework for retesting and to clarify how these results should influence subsequent treatment decisions.
Quality measures for care of patients with restless legs syndrome: 2025 update after measure maintenance
Lloyd, Robin M. , Crawford, T’Auna, Donald, Ryan, Gray, Diedra D., Junna, Mithri R. , Lewin, Daniel , Revana, Amee , Schutte-Rodin, Sharon, Healy, William J.

Journal of Clinical Sleep Medicine | September 17, 2025 | Volume: 21 | Issue: 12 | Pages: 2205-2211

10.5664/jcsm.11894 | PMID 40958636 Get It

No Difference in Clinical Outcomes Between Concomitant Anterior and Posterior Cruciate Ligament Reconstruction With and Without Independent Suture Tape Augmentation
Tagliero, Adam J. , Cabarcas, Brandon C. , Iyer, Sanathan, Daniel, Adam V. , Kelly, John J., Krych, Aaron J. , Levy, Bruce A., Cabarcas, Brandon , Iyer, Sanathan , Krych, Aaron J , Tagliero, Adam , Iyer, Sanathan, Levy, Bruce A.

Arthroscopy, Sports Medicine, and Rehabilitation | September 17, 2025 | Volume: 7 | Issue: 6 | Pages: No page available

10.1016/j.asmr.2025.101261 | PMID 41541520 Get It

Purpose To compare clinical and functional outcomes (including range of motion, clinical laxity, reoperation/revision rates, and patient‐reported outcome measures) of combined anterior cruciate ligament/posterior cruciate ligament reconstruction (ACLR/PCLR) with and without suture tape (ST) augmentation via a propensity‐matched cohort analysis. Methods A retrospective review was performed to identify all patients who underwent simultaneous primary ACLR/PCLR between 2001 and 2022. A 1:1 propensity match was performed using patient sex, Schenck classification grade, age ±8 years, and body mass index ±10 to produce 2 cohorts: ACLR/PCLR with and without (control) ST augmentation. Clinical outcomes (visual analog scale [VAS], range of motion, Tegner activity scale, Lysholm score, and International Knee Documentation Committee subjective score) were analyzed. Results The propensity match produced a final cohort of 50 ACLR/PCLR patients: 25 with ST augmentation and 25 nonaugmented controls. Most patients had a KD3M injury (12 in each cohort), and no significant differences were noted in concomitant neurovascular, meniscal, or chondral injuries between the groups. Forty patients (80%) completed follow‐up at a mean of 4.7 years for the ST cohort and 8.5 years for the control cohort ( P > .05). Postoperative ST versus control values for outcome measures including VAS at rest (0.9 vs 1.5; P = .71), VAS with use (3.0 vs 2.2; P = .24), International Knee Documentation Committee (75.7 vs 76.3; P = .37), Lysholm (81.8 vs 77.6; P = .91), and Tegner activity (4.8 vs 5.4; P = .37) scores showed no differences ( P > .05 for all). There were no differences in excessive postoperative clinical laxity between ST and control cohorts (ST n = 1 vs control n = 2, Lachman 2+, P = .140; ST n = 3 vs control n = 1, posterior drawer 2+, P = .122). The total number of patients who underwent reoperation was 7 of 25 (28%) for the ST cohort and 8 of 25 (32%) for the control cohort ( P = .76). Conclusions Concomitant ACLR/PCLR utilizing ST augmentation showed similarly satisfactory postoperative clinical outcomes at a minimum 2‐year follow‐up compared to a matched cohort without ST. The use of an independent ST did not limit motion or increase the incidence of complications or revision surgery. Level of Evidence Level III, retrospective comparative case series.
Everolimus and Low-Dose Tacrolimus After Heart Transplant in Children
Almond, Christopher S. , Alejos, Juan , Ameduri, Rebecca , Barkoff, L., Barnes, Aliessa, Butto, Arene , Carlo, Waldemar F. , Chrisant, Maryanne , Dreyer, William J. , Everitt, Melanie D. , Gonzales, S., Hollander, Seth A. , Kindel, Steven J. , Klein, Gloria L., Lamour, Jacqueline M. , Lee, Joanne , Lü, Minmin , Lytrivi, Irene D. , Miyamoto, Shelley D. , Pahl, Elfriede , Peng, David M. , Sutcliffe, David , Ybarra, A., Zangwill, Steven , Rossano, Joseph W. , Investigators, TEAMMATE Trial, Choi, Jua , Wilber, Jared, Sutherland, Scott M. , Feng, Dana, Lagerborg, Kendra, Burns, Julia, Nikolli, Kledja, Twombly, Adrianna, Massey, Linda K., Wartick, Jennifer, Yohannes, Kifle, Gupta, Akanksha , Major, Melissa , Blume, Elizabeth D. , Esteso, Paul , VanderPluym, Christina , Bastardi, Heather , MacLean, Matthew T. , Haschke, Paul, Gibbons, Taylor , Vu, Courtney , Reilly, Sydney, Ha, Lynne, Boyle, Carley, Robie, Sharon L., Ogata, Kay, Knecht, Carlos, Browne, Lorna P. , Malone, LaDonna J. , Gordon, Megyn, Molina, Kimberly , Cabrera, Antonio G. , Lambert, Linda, Fullerton, Chase, Thompson, Kate J., Cardon, Michelle, Timmer, Amanda, Nelson, Michelle , Ramstack, Dani, Heyrend, Carly, Benefield, Emily C., Roberson, Teresa, Butts, Ryan J. , Martinez, Maria , Pottorre, Simi, Burgardt, Laura, Armstrong, Blake, Zamberlan, M. , McCormick, Amanda D. , Bartsch, Jaynee, Mahle, William T. , Burnett, Meilssa, Pietra, Biagio A. , Breault, Leah, Friedland‐Little, Joshua , Prakash, Ranjini, Lee, Anne CC , Brody, Linnea, Huberts, Avery, Noon, Kelli, Guadiz, Donna, Garcia, Allison, Addonizio, Linda J. , Repp, Arielle, Cornelius, C.E. , Abraham, Margaret, Rose-Felker, Kristen, Moninger, Allison, Huston, Allison, Berman, Pamela, Ploutz, Michelle , Wisotzkey, Bethany , Grohall, Stacey, Colon, April, Churosh, Jenny, Cordova, Renee, Smith, Paulette, Kornetsky, Susan , Mitchell, Anna, Alderson, Daniel, Dominguez, R Blanco, Gawlowicz, Kennan, Lipsitz, Stuart R. , Eisen, Howard J. , Jeewa, Aamir , Uber, Patricia A., Thakar, Rahul, Ellsworth, Darrell L. , McCormick, A , Peng, D M , Daly, Kevin P , Albers, Erin L. , Auerbach, Scott R. , Bock, Matthew J. , Castleberry, Chesney , Deshpande, Shriprasad R. , Feingold, Brian , Lal, Ashwin K. , Ryan, Thomas D. , Singh, Tajinder P. , Su, Jennifer A. , Sleeper, Lynn A. , Investigators, TEAMMATE Trial, Wilber, Jared, Nikolli, Kledja, Massey, Linda, Wartick, Jennifer, Major, Melissa, MacLean, Matthew, Lee, JungWoo, Joong, Anna , Reilly, Sydney, Kuhn, Michael, Ogata, Kay, Knecht, Carol, Gordon, Megyn, Hansen, Lisa, May, Lindsay J. , Lambert, Linda, Fullerton, Chase, Thompson, Kate, Timmer, Amanda, Heyrend, Carly, Martinez, Maria, Schumacher, Kurt R. , Lim, Heang M. , Bartsch, Jaynee, Burnett, Meilssa, Prakash, Ranjini, Lee, Anne CC , Brody, Linnea, Huberts, Avery, Noon, Kelli, Guadiz, Donna, Repp, Arielle, Bansal, Neha , Hsu, Daphne T. , Cornelius, Chelsea, Padilla, Luz A. , Churosh, Jenny, Cordova, Renee, Mitchell, Anna, Alderson, Daniel, Dominguez, Robleinscky, KOBASHIGAWA, JON A. , Wightman, Aaron G.

JAMA | September 17, 2025 | Volume: 334 | Issue: 15 | Pages: 1339

10.1001/jama.2025.14338 | PMID 40960806 Get It

ImportanceStudies suggest that everolimus may reduce the risk of rejection, cardiac allograft vasculopathy (CAV), chronic kidney disease (CKD), and cytomegalovirus (CMV) after heart transplant. Everolimus use is controversial because of data demonstrating higher infection deaths when everolimus is introduced de novo after transplant. It is unclear whether everolimus is safe and effective when initiated at 6 months posttransplant in children, a population in which median graft survival is limited to 15 years and randomized clinical trials are lacking.ObjectiveTo evaluate the safety and efficacy of everolimus combined with low-dose tacrolimus to prevent major adverse transplant events (MATEs) in children after heart transplant.Design, Setting, and ParticipantsMulticenter, randomized, open-label, clinical trial enrolling 211 patients who were alive 6 months after pediatric heart transplant at 25 US sites from February 2018 to August 2020. The last date of follow-up was April 17, 2023.InterventionsParticipants were randomized to receive everolimus and low-dose tacrolimus (n = 107) or standard-dose tacrolimus and mycophenolate mofetil (n = 104) for 30 months.Main Outcomes and MeasuresThe primary efficacy end point was the MATE-3 score at 30 months, a validated composite ordinal end point including acute cellular rejection, CAV, and CKD. The primary safety end point was the MATE-6 score, encompassing the MATE-3 score plus antibody-mediated rejection, infection, and posttransplant lymphoproliferative disorder.ResultsAmong 211 children randomized, the mean age was 8.2 (SD, 6.3) years, 97 (46%) underwent transplant for congenital heart disease, and 49 (23%) were treated for rejection before 6 months. At 30 months, the mean MATE-3 score did not differ between the 2 treatment groups (mean difference, −0.32; 95% CI, −0.90 to 0.20; P = .16). The mean MATE-6 score was no higher in the everolimus group than in the mycophenolate group (baseline-adjusted mean difference, −0.40; 95% CI, −1.81 to 0.93), meeting the success criterion for safety (noninferiority margin &amp;lt;3). There were no differences in graft survival, MATE-free survival, or freedom from any individual MATE. Everolimus was associated with greater improvement in estimated glomerular filtration rate at 12 months (mean difference, 10.5 mL/min/1.73 m2; 95% CI, 1.09-19.91 mL/min/1.73 m2) and a lower incidence of CMV infection (hazard ratio, 0.50; 95% CI, 0.26-0.93).Conclusions and RelevanceAmong 6-month pediatric heart transplant survivors, everolimus and low-dose tacrolimus did not differ from tacrolimus and mycophenolate in preventing the composite of cellular rejection, CAV, and CKD at 30 months. However, everolimus and low-dose tacrolimus appear to be safe based on the total burden of 6 MATEs and may be associated with improved kidney function and less CMV infection.Trial RegistrationClinicalTrials.gov Identifier: NCT03386539