tbuckley/cabot-search huggingface dataset:
Heterogeneity of cell composition and origin identified by single-cell transcriptomics in renal cysts of patients with autosomal dominant polycystic kidney disease
Authors: Qiong Li, Yuchen Wang, Wenfeng Deng, Yanna Liu, Jian Geng, Ziyan Yan, Fei Li, Binshen Chen, Zhuolin Li, Renfei Xia, Wenli Zeng, Rumin Liu, Jian Xu, Fu Xiong, Chin-Lee Wu, Yun Miao
Journal: Theranostics (2021)

Rationale: Renal cysts in patients with autosomal dominant polycystic kidney disease (ADPKD) can originate from any nephron segments, including proximal tubules (PT), the loop of Henle (LOH), distal tubules (DT), and collecting ducts (CD). Previous studies mostly used limited cell markers and failed to identify cells negative for these markers. Therefore, the cell composition and origin of ADPKD cyst are still unclear, and mechanisms of cystogenesis of different origins await further exploration. Methods: We performed single-cell RNA sequencing for the normal kidney tissue and seven cysts derived from superficial or deep layers of the polycystic kidney from an ADPKD patient. Results: Twelve cell types were identified and analyzed. We found that a renal cyst could be derived either from CD or both PT and LOH. Gene set variation analysis (GSVA) showed that epithelial mesenchymal transition (EMT), TNFA signaling via the NFKB pathways, and xenobiotic metabolism were significantly activated in PT-derived cyst epithelial cells while robust expression of genes involved in G2M Checkpoint, mTORC1 signaling, E2F Targets, MYC Targets V1, MYC Targets V2 were observed in CD-derived cells. Conclusion: Our results revealed that a single cyst could originate from CD or both PT and LOH, suggesting heterogeneity of polycystic composition and origin. Furthermore, cyst epithelial cells with different origins have different gene set activation.
Assessing the Impact of Prophylactic Anidulafungin during Remission Induction of Acute Myeloid Leukemia - a Propensity-Score Matching Analysis
Authors: Wellington F Silva, Fernanda Rodrigues Mendes, Raphael Bandeira Melo, Elvira Velloso, Vanderson Rocha, Eduardo M Rego
Journal: Blood (2021)
Abstract 14544: Cardiovascular Outcomes Among Patients With Acute Coronary Syndromes and Diabetes in India: Results From ACS QUIK Trial
Authors: Abdulhamied Alfaddagh, Haitham Khraishah, Mohamad Kassab, Zeb McMillan, Mazen Albaghdadi
Journal: Circulation (2021)

Introduction: Patients with diabetes are more likely to experience an acute myocardial infarction (AMI), heart failure, or death from cardiovascular (CV) causes than patients without diabetes. Despite cardiovascular disease being the leading cause of death in India, limited data exists on the outcome of patients with diabetes who suffer acute cardiovascular events. Hypothesis: Diabetes is common in patients with acute myocardial infarction (AMI) in India and is associated with worse in-hospital and 30-day cardiovascular outcomes. Methods: We examined 21374 patients with AMI enrolled in the Acute Coronary Syndrome Quality Improvement in Kerala trial. We compared those with and without diabetes with regards to the development of in-hospital and 30-day major adverse cardiac events including death, re-infarction, stroke, or major bleeding. The associations between diabetes and cardiac outcomes were adjusted for presentation and in-hospital management using logistic regression. Results: mean±SD age was 60.1±12.0 years and 24.3% were females. A total of 44.4% of AMI patients had diabetes. Those with diabetes were more likely to be older, female, with hypertension and have higher Killip class on presentation but less likely to present with STEMI. Patients with diabetes had longer symptoms onset-to-arrival (median 225 vs 290 min; P&lt;0.001) and, in case of STEMI, longer door-to-balloon times (median, 75.0 vs 91 min; P&lt;0.001). Diabetes was independently associated with higher in-hospital death (adjusted odds ratio [aOR], 1.46; 95% CI, 1.12-1.89), in-hospital reinfarction (aOR 1.52; 95% CI, 1.15-2.02). Diabetes was also associated with higher 30-day MACE (aOR, 1.33; 95% CI, 1.14-1.55) and 30-day death (aOR, 1.40; 95%CI, 1.16-1.69) but not 30-day stroke or 30-day bleeding. Conclusion: Among patients presenting with AMI in Kerala, India, a considerable proportion has diabetes and are at increased risk for in-hospital and 30-day adverse cardiovascular outcomes. Increased awareness of the increased CV risk and attention to the implementation of established cardiovascular therapies are indicated for patients with diabetes in lower-middle income countries who develop AMI.
Preclinically Effective Menin Inhibitor SNDX-50469 and SNDX-5613-Based Combinations Against MLL1-Rearranged (MLL-r) or NPM1-Mutant AML Models
Authors: Warren C. Fiskus, Christopher Peter Mill, Christine Birdwell, John A. Davis, Arnold Salazar, Kevin Philip, Tapan M. Kadia, Courtney D. DiNardo, Koichi Takahashi, Gerard M. McGeehan, Naval Daver, Kapil N. Bhalla
Journal: Blood (2021)
Clonal Tracking By Whole Genome Sequencing Permits Comprehensive Mapping of the Genomic Landscape in Pre- and Post-Gene Therapy Sickle Cell Patients
Authors: Alyssa H. Cull, Michael Spencer Chapman, Marioara Ciuculescu, Emily Mitchell, Myriam Armant, Grace Boyd, Erica B. Esrick, David A. Williams, Peter J. Campbell, David G. Kent
Journal: Blood (2021)
Abstract 14246: Prognostic Implication of Elevated Troponin in Patients Visiting Emergency Department Without Diagnosis of Specific Cardiovascular Disease
Authors: Minseok Song, Jin-Ho Choi, Taerim Kim, Jongen Park, Soohyun Park
Journal: Circulation (2019)

Background: Elevation of cardiac troponin is not uncommon in patients visiting emergency department without specific coronary artery disease. The prognostic implication of elevated troponin in thes...
VEGF pathway inhibition potentiates PARP inhibitor efficacy in ovarian cancer independent of BRCA status
Authors: Francesca Bizzaro, Ilaria Fuso Nerini, Molly A. Taylor, Alessia Anastasia, Massimo Russo, Giovanna Damia, Federica Guffanti, Francesca Guana, Paola Ostano, Lucia Minoli, Maureen M. Hattersley, Stephanie Arnold, Antonio Ramos-Montoya, Stuart C. Williamson, Alessandro Galbiati, Jelena Urosevic, Elisabetta Leo, Ugo Cavallaro, Carmen Ghilardi, Simon T. Barry, Maria Rosa Bani, Raffaella Giavazzi
Journal: Journal of Hematology & Oncology (2021)

Abstract Poly ADP-ribose polymerase inhibitors (PARPi) have transformed ovarian cancer (OC) treatment, primarily for tumours deficient in homologous recombination repair. Combining VEGF-signalling inhibitors with PARPi has enhanced clinical benefit in OC. To study drivers of efficacy when combining PARP inhibition and VEGF-signalling, a cohort of patient-derived ovarian cancer xenografts (OC-PDXs), representative of the molecular characteristics and drug sensitivity of patient tumours, were treated with the PARPi olaparib and the VEGFR inhibitor cediranib at clinically relevant doses. The combination showed broad anti-tumour activity, reducing growth of all OC-PDXs, regardless of the homologous recombination repair (HRR) mutational status, with greater additive combination benefit in tumours poorly sensitive to platinum and olaparib. In orthotopic models, the combined treatment reduced tumour dissemination in the peritoneal cavity and prolonged survival. Enhanced combination benefit was independent of tumour cell expression of receptor tyrosine kinases targeted by cediranib, and not associated with change in expression of genes associated with DNA repair machinery. However, the combination of cediranib with olaparib was effective in reducing tumour vasculature in all the OC-PDXs. Collectively our data suggest that olaparib and cediranib act through complementary mechanisms affecting tumour cells and tumour microenvironment, respectively. This detailed analysis of the combined effect of VEGF-signalling and PARP inhibitors in OC-PDXs suggest that despite broad activity, there is no dominant common mechanistic inter-dependency driving therapeutic benefit.
In Utero Transplantation of FVIII-Expressing Human Placental Cells Upregulates Gene Pathways Associated with Immune Tolerance, Altering the Response to Postnatal FVIII Infusion
Authors: Martin Rodriguez, Brady Trevisan, Sunil George, Jordan E Shields, Jorge Figueroa, Diane Meares, John Owen, Anthony Atala, Christopher B Doering, H. Trent Spencer, Christopher D Porada, M. Graca Almeida-Porada
Journal: Blood (2021)
HMGA1 Chromatin Regulators Drive Progression in Myeloproliferative Neoplasms through Epigenetic Rewiring to Induce Networks Involved in GATA2 and Proliferation
Authors: Liping Li, Jung-Hyun Kim, Wenyan Lu, Leslie Cope, Donna M Williams, Joseph Kim, Raajit Rampal, Richard Koche, Lingling Xian, Li Luo, Marija Vasiljevic, Daniel R Matson, Zhizhuang Joe Zhao, Ophelia Rogers, Matthew C. Stubbs, Karen Reddy, Jerry L. Spivak, Alison R. Moliterno, Linda Resar
Journal: Blood (2021)
Mean Versus Peak Coronary Calcium Density on Non-Contrast CT
Authors: Omar Dzaye, Alexander C. Razavi, Zeina A. Dardari, Daniel S. Berman, Matthew J. Budoff, Michael D. Miedema, Olufunmilayo H. Obisesan, Ellen Boakye, Khurram Nasir, Alan Rozanski, John A. Rumberger, Leslee J. Shaw, Martin Bødtker Mortensen, Seamus P. Whelton, Michael J. Blaha
Journal: JACC. Cardiovascular imaging (2021)
Early Administration of Partially HLA Matched Third Party Virus-Specific T-Cells in Conjunction with Antiviral Treatment for Initial Viral Infection after Allogeneic Stem Cell Transplant Is Safe and Leads to High Rates of Viral Control
Authors: Wei Jiang, Gaurav Sutrave, Selmir Avdic, Leighton Edward Clancy, Shafqat Inam, Elissa Atkins, Caroline M Bateman, Adrian Gabriel Selim, Vicki Antonenas, Janine Street, Renee Simms, Peter J. Shaw, David Ritchie, Emily Blyth, David J Gottlieb
Journal: Blood (2021)
Detection of Double Hit and Triple Hit Cytogenetics at Relapse Identifies a Very High Risk Subset of Relapsed/Refractory Multiple Myeloma Patients
Authors: Charanpreet Singh, Sreejesh Sreedharanunni, Vandana Panakkal, Aditya Jandial, Arihant Jain, Deepesh Lad, Gaurav Prakash, Alka Khadwal, Pankaj Malhotra
Journal: Blood (2021)
First Results of the Risk-Adapted, MRD-Stratified GMALL Trial 08/2013 in 705 Adults with Newly Diagnosed Acute Lymphoblastic Leukemia/Lymphoma (ALL/LBL)
Authors: Nicola Goekbuget, Matthias Stelljes, Andreas Viardot, Kathrin Nachtkamp, Björn Steffen, Folker Schneller, Nael Alakel, Max Topp, Boris Böll, Christoph Faul, Karsten Spiekermann, Knut Wendelin, Maher Hanoun, Ralph Wäsch, Joachim Beck, Sonja Martin, Vladan Vucinic, Claudia D. Baldus, Monika Brüggemann, Thomas Burmeister, Heike Pfeifer, Stefan Schwartz, Lena Baumann, Diana Tichy, Hubert Serve, Walter Fiedler
Journal: Blood (2021)
Safety and immunogenicity of a SARS-CoV-2 inactivated vaccine in patients with chronic hepatitis B virus infection
Authors: Tiandan Xiang, Boyun Liang, Hua Wang, Xufeng Quan, Shengsong He, Helong Zhou, Yongwen He, Dongliang Yang, Baoju Wang, Xin Zheng
Journal: Cellular and Molecular Immunology (2021)
An MMP-degradable and conductive hydrogel to stabilize HIF-1α for recovering cardiac functions
Authors: Xiaojuan Wei, Si Chen, Tian Xie, Hongchi Chen, Xin Jin, Jumin Yang, Shafaq Sahar, Huanlei Huang, Shuoji Zhu, Nanbo Liu, Changjiang Yu, Ping Zhu, Wei Wang, Wei Zhang
Journal: Theranostics (2021)

Rationale: Although a few injectable hydrogels have shown a reliable biosafety and a moderate promise in treating myocardial infarction (MI), the updated hydrogel systems with an on-demand biodegradation and multi-biofunctions to deliver therapeutic drug would achieve more prominent efficacy in the future applications.In this report, a conductive and injectable hydrogel crosslinked by matrix metalloproteinasesensitive peptides (MMP-SP) was rationally constructed to stabilize hypoxia-inducible factor-1α (HIF-1α) to recover heart functions after MI.Methods: Firstly, tetraaniline (TA) was incorporated into partially oxidized alginate (ALG-CHO) to endow the hydrogels with conductivity.The 1,4-dihydrophenonthrolin-4-one-3-carboxylic acid (DPCA) nanodrug was manufactured with high drug loading capacity and decorated with polymerized dopamine (PDA) to achieve a stable release of the drug.Both ALG-CHO and DPCA@PDA can be cross-linked by thiolated hyaluronic acid (HA-SH) and thiolated MMP-SP to construct a MMP-degradable and conductive hydrogel.After administration in the infarcted heart of rats, echocardiographic assessments, histological evaluation, and RT-PCR were used to evaluate therapeutic effects of hydrogels.Results: The cell viability and the results of subcutaneous implantation verify a good cytocompatibility and biocompatibility of the resulting hydrogels.The hydrogel shows remarkable strength in decreasing the expression of inflammatory factors, maintaining a high level of HIF-1α to promote the vascularization, and promoting the expression of junctional protein connexin 43.Meanwhile, the multifunctional hydrogels greatly reduce the infarcted area (by 33.8%) and improve cardiac functions dramatically with ejection fraction (EF) and fractional shortening (FS) being increased by 31.3% and 19.0%, respectively. Conclusion:The as-prepared hydrogels in this report achieve a favorable therapeutic effect, offering a promising therapeutic strategy for treating heart injury.
The Selective Bruton Tyrosine Kinase (BTK) Inhibitor TG-1701 As Monotherapy and in Combination with Ublituximab and Umbralisib (U2) in Patients with B-Cell Malignancies
Authors: Chan Yoon Cheah, Wojciech Jurczak, Masa Lasica, Tomasz Wróbel, Stanley Cheung, Jan Walewski, Krzysztof Giannopoulos, Costas K. Yannakou, Katharine L Lewis, Monika Dlugosz-Danecka, Hari P. Miskin, Alejandro D. Ricart, Owen A. O'Connor, Constantine S. Tam
Journal: Blood (2021)
Abstract 9380: Rescuing Mitochondrial Fusion/Fission Balance Mitigates Oxidative Stress, Myocardial Injury and Apoptosis in Rats with Doxorubicin-induced Cardiotoxicity
Authors: Chayodom Maneechote, Thawatchai Khuanjing, Benjamin Ongnok, Apiwan Arinno, Nanthip Prathumsap, Titikorn Chunchai, Busarin Arunsak, Sasiwan Kerdphoo, Siriporn Chattipakorn, Nipon Chattipakorn
Journal: Circulation (2021)

Introduction: Doxorubicin (DOX) has been an extensively used chemotherapeutic drug in handling a wide range of cancers. Cardiotoxicity induced by DOX is a serious adverse effect of oncologic therapy. Although mitochondrial dynamic modulators including mitochondrial fission inhibitor (Mdivi-1) and fusion promoter (M1) have been shown as potential therapeutic remedies to treat various cardiovascular disorders, the cardioprotective effects of Mdivi-1 and M1 in DOX-induced cardiotoxicity is not known. Hypothesis: Mdivi-1 and M1 treatments reduce oxidative stress, myocardial injury, and programmed cell death by apoptosis, leading to improved left ventricular (LV) function in rats with DOX-induced cardiotoxicity. Methods: Male Wistar rats (n=20) were injected with either DOX (3 mg/kg, 6 doses, n=5) or 3% DMSO in NSS (n=5) as a control. In the DOX group, DOX-treated rats were received one of the following treatments: 1) 3% DMSO in NSS (n=5), 2) Mdivi-1 (1.2 mg/kg/day, n=5), 3) M1 (2 mg/kg/day, n=5). All treatments were given to the rats via intraperitoneal injection for 30 days beginning simultaneously with the first DOX injection. LV function, cardiac tissue malondialdehyde (MDA), serum cardiac troponin-I (c-TnI), and cleaved caspase-3 (CC3) protein levels were determined. Results: Injection of DOX markedly increased cardiac MDA, c-TnI, and CC3 levels (Fig. 1A-D). Impaired left ventricular ejection fraction (67% for DOX vs. 91% for control) was observed. Mdivi-1 and M1 co-treatment remarkably reduced the levels of MDA, c-TnI, and CC3, leading to increased LVEF from 67% in DOX to 89% in Mdivi-1 and 90% in M1, respectively (Fig. 1A-D). Conclusions: Mitochondrial fission inhibitor and fusion promoter effectively protected the heart from DOX-induced cardiotoxicity through suppression of oxidative stress, myocardial injury, and apoptosis. These findings indicate the mitochondrial dynamics as novel therapeutic targets in preventing Dox-induced cardiotoxicity.
Hereditary Chronic Neutrophilic Leukemia in a Four Generation Family without Transformation to Acute Leukemia
Authors: Zane Chiad, Amanda Lance, Sara L. Seegers, Sarah-Catherine Paschall, Kendra Drummond, Nury M Steuerwald, Peter M. Voorhees, Belinda Avalos, Lawrence J Druhan
Journal: Blood (2021)
A Multi-Center Retrospective Study of Polatuzumab for Patients with Large B-Cell Lymphoma Relapsed after Standard of Care CAR T-Cell Therapy
Authors: Sushanth Gouni, Allison C. Rosenthal, Jennifer L. Crombie, Andrew Ip, Manali Kamdar, Brian T. Hess, Lei Feng, Grace Watson, Amy Ayers, Sattva S. Neelapu, Arushi Khurana, Madiha Iqbal, Yi Lin, Reid W Merryman, Paolo Strati
Journal: Blood (2021)
Brentuximab Vedotin As Consolidation Therapy Following Autologous Stem Cell Transplantation in Children and Adolescents with Relapsed/Refractory Hodgkin Lymphoma: A Multi-Center Retrospective Analysis
Authors: Christopher J. Forlenza, Jaclyn Rosenzweig, Audrey Mauguen, Ilia Buhtoiarov, Branko Cuglievan, Hema Dave, Rebecca J Deyell, Jaime Flerlage, Anna Franklin, Jennifer Krajewski, Kasey J Leger, Lianna Marks, Robin Norris, Martha Pacheco, Faye Willen, Adam Paul Yan, Paul Harker-Murray, Lisa Giulino Roth
Journal: Blood (2021)
Whole Genome Sequencing of Diamond Blackfan Anemia Syndrome Patients Detects Mutations That Alter mRNA Splicing
Authors: Stanley R Clarke, Adrianna Vlachos, Jens Lichtenberg, Nancy E Seidel, Jaya Jagadeesh, NISC Comparative SequencingProgram, Adam M. Phillippy, Jason E Farrar, Steven R Ellis, Irma Dianzani, Jeffrey M Lipton, David M Bodine
Journal: Blood (2021)
In Africa, a High Proportion of Adults with HbSC Meet American Society of Hematology's Eligibility Criteria for Severe Sickle Cell Disease and Starting Hydroxyurea Therapy in a Clinical Trial Setting
Authors: William Kwesi Ghunney, Eugenia Vicky Asare, John Ayete-Nyampong, Yvonne Adomakoh, Adetola A. Kassim, Samuel A Oppong, Mark Rodeghier, Michael R. DeBaun, Edeghonghon Olayemi
Journal: Blood (2021)
Selective identification and localization of indolent and aggressive prostate cancers via CorrSigNIA: an MRI-pathology correlation and deep learning framework
Authors: Indrani Bhattacharya, Arun Seetharaman, Christian Kunder, Wei Shao, Leo C. Chen, Simon J.C. Soerensen, Jeffrey B. Wang, Nikola C. Teslovich, Richard E. Fan, Pejman Ghanouni, James D. Brooks, Geoffrey A. Sonn, Mirabela Rusu
Journal: Medical Image Analysis (2021)

Automated methods for detecting prostate cancer and distinguishing indolent from aggressive disease on Magnetic Resonance Imaging (MRI) could assist in early diagnosis and treatment planning. Existing automated methods of prostate cancer detection mostly rely on ground truth labels with limited accuracy, ignore disease pathology characteristics observed on resected tissue, and cannot selectively identify aggressive (Gleason Pattern≥4) and indolent (Gleason Pattern=3) cancers when they co-exist in mixed lesions. In this paper, we present a radiology-pathology fusion approach, CorrSigNIA, for the selective identification and localization of indolent and aggressive prostate cancer on MRI. CorrSigNIA uses registered MRI and whole-mount histopathology images from radical prostatectomy patients to derive accurate ground truth labels and learn correlated features between radiology and pathology images. These correlated features are then used in a convolutional neural network architecture to detect and localize normal tissue, indolent cancer, and aggressive cancer on prostate MRI. CorrSigNIA was trained and validated on a dataset of 98 men, including 74 men that underwent radical prostatectomy and 24 men with normal prostate MRI. CorrSigNIA was tested on three independent test sets including 55 men that underwent radical prostatectomy, 275 men that underwent targeted biopsies, and 15 men with normal prostate MRI. CorrSigNIA achieved an accuracy of 80% in distinguishing between men with and without cancer, a lesion-level ROC-AUC of 0.81±0.31 in detecting cancers in both radical prostatectomy and biopsy cohort patients, and lesion-levels ROC-AUCs of 0.82±0.31 and 0.86±0.26 in detecting clinically significant cancers in radical prostatectomy and biopsy cohort patients respectively. CorrSigNIA consistently outperformed other methods across different evaluation metrics and cohorts. In clinical settings, CorrSigNIA may be used in prostate cancer detection as well as in selective identification of indolent and aggressive components of prostate cancer, thereby improving prostate cancer care by helping guide targeted biopsies, reducing unnecessary biopsies, and selecting and planning treatment.
Estimating burden of syphilis among men who have sex with men
Authors: Ting-Ting Jiang, Ning-Xiao Cao, Xiang-Sheng Chen
Journal: The Lancet Global Health (2021)

In their Article in The Lancet Global Health, Motoyuki Tsuboi and colleagues1 described the burden of syphilis among men who have sex with men (MSM) in different sustainable development goal regions, on the basis of a global systematic review and meta-analysis of published studies done between Jan 1, 2000, and Feb 1, 2020. In their analysis, the authors estimated a global pooled syphilis prevalence of 7·5% (95% CI 7·0–8·0) between 2000 and 2020 using 345 data points consisting of the 606 232 MSM tested, and the pooled prevalence in 2010–20 (6·6%, 6·0–7·2, 200 data points; n=420 355) was significantly lower than that of 2000–09 (8·9%, 8·0–9·9, 145 data points; n=185 877; p<0·0001).
Abstract 10969: Estimated Number Needed to Treat with Mavacamten vs Placebo to Improve Functional Capacity and Left Ventricular Outflow Tract Obstruction in Patients with Symptomatic Obstructive Hypertrophic Cardiomyopathy
Authors: Marc Semigran, Yan Wang, Jipan Xie, Yaping Xu, Megan B Sutton, Nihar R Desai
Journal: Circulation (2021)

Introduction: Mavacamten improved functional capacity and LVOT obstruction in patients with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in the EXPLORER-HCM study (NCT03470545). Objective: To determine the number needed to treat (NNT) to achieve a clinical response comparing 30-week treatment with mavacamten versus placebo in patients with symptomatic oHCM. Methods: Data from the EXPLORER-HCM trial were analyzed. Most patients (92%) were receiving beta-blockers or calcium channel blockers. Responses were assessed using several measures: primary efficacy endpoint (improvement of ≥1.5 mL/kg/min in pVO 2 and improvement of ≥1 NYHA class or improvement of ≥3.0 mL/kg/min in pVO 2 and no worsening in NYHA class); symptoms (NYHA class); PROs (Kansas City Cardiomyopathy Questionnaire Clinical Summary Score [KCCQ CSS] and Hypertrophic Cardiomyopathy Symptom Questionnaire Shortness of Breath [HCMSQ SoB]); hemodynamics (resting and post-exercise LVOT gradients); exercise capacity (pVO 2 ); biomarkers (NT-proBNP and hs-cTnI). NNT comparing mavacamten versus placebo was calculated as the reciprocal of the difference in response rates between the two groups at week 30 rounded up to an integer. Results (Table): The NNT to achieve the primary endpoint was 6. The NNTs were 3 for an improvement of ≥1 NYHA class and 4 for an improvement to NYHA class I. For PROs, the NNTs were 5 for an increase of ≥10 points in KCCQ CSS and 4 for a reduction of ≥2.5 points in HCMSQ SoB, which are considered clinically important changes. For hemodynamics, the NNTs were 2–3. For increases in pVO 2 ≥1.5 mL/kg/min and ≥3.0 mL/kg/min, the NNTs were 6 and 9, respectively. For biomarkers, the NNTs were 3 for any reduction in NT-proBNP and 5 for any reduction in hs-cTnI. Conclusions: The NNTs comparing mavacamten with placebo were consistently low across several clinical and PRO measures. This suggests that mavacamten may be an effective therapy for the treatment of symptomatic oHCM.