Hematology Commons^™

Relationships Between Health Behaviors And Markers Of Oxidative Stress And Inflammation On Blood Pressure, Alison M. Godfrey

Honors Scholar Theses

Background. Young adulthood is a period when cardiometabolic risk factors begin to emerge. Developing cardiometabolic risk early can increase the risk and severity of chronic illness across the lifespan. Despite this, young adults are often understudied particularly as it relates to major cardiometabolic risk factors such as blood pressure (BP) and risk of hypertension. Hypertension is a public health crisis and early indicators of hypertension risk should be more thoroughly researched. Therefore, the purpose of the presented thesis is to examine BP in young adults and two potential pathways by which risk may emerge in young adults; inflammation (C-Reactive …

Evaluating The Therapeutic Potential Of Geranylgeranyl Diphosphate Synthase Inhibitor, Ram2061, In Myeloma Bone Disease, Molly Muehlebach

Theses & Dissertations

Multiple myeloma (MM) is a hematological malignancy of the plasma cells which often results in lytic bone disease. Myeloma bone disease (MBD), characterized by diffuse osteopenia, bone lesions, or pathological fracture, results from MM mediated promotion of osteoclast resorption and dual inhibition of bone formation, drastically reducing bone strength and integrity. Development of MBD is known to significantly impact patient quality of life and increase risk of mortality. Considering that up to 80-95% of MM patients are expected to develop bone disease post-initial MM diagnosis, there is a significant need within this population for therapeutics which address both MM disease …

Can Anemia Be A Prognostic Indicator To Scope For Gastroesophageal Junction Adenocarcinoma?, Ryan Tam, Neha Narayanan, Evan Basha, Joel Thompson

Advances in Clinical Medical Research and Healthcare Delivery

Gastroesophageal junction adenocarcinoma is a rapidly progressive disease that has a poor prognosis with a 5-year survival rate of 20%. It commonly presents with major symptoms of dysphagia and weight loss in addition to a long-standing history of reflux. As of now, screening for esophageal adenocarcinoma (EAC) is dependent on identifying risk factors which include a family history of Barrett’s esophagus and esophageal adenocarcinoma or patients with gastroesophageal reflux disease and at least one other risk factory for EAC such as age greater than 50 years, obesity or central adiposity, history of smoking, or male gender. Here, we present a …

Mystery Of Maha: Hypertension’S Twist Over Cancer’S Shadow, Andrew Takla, Gabriel Vachon, John Hix, Nagesh Jadhav

Advances in Clinical Medical Research and Healthcare Delivery

Microangiopathic hemolytic anemia (MAHA) refers to nonimmune hemolysis resulting from the intravascular fragmentation of red blood cells. The etiology of such disorders can range from primary thrombotic microangiopathic syndromes (TMA) to various systemic conditions, posing a diagnostic challenge. Prompt identification requires a sharp clinical assessment to improve outcomes.

Iron Deficiency—More Than Just Anemia: A Literature Review, Arianna S Moss, Zahra Pakbaz

Journal of Community Hospital Internal Medicine Perspectives

Iron deficiency (ID) is a global public health concern, as it can be a prodrome of gastrointestinal (GI) malignancies, exacerbate chronic conditions, and lead to an overall decreased quality of life. Despite being the most prevalent nutritional deficiency, it remains underdiagnosed by many healthcare professionals. Misconceptions regarding the clinical and laboratory manifestations of ID lead to delayed or missed diagnoses, contributing to poor patient outcomes. Though anemia is a commonly known consequence of ID, there are additional consequences that remain underrecognized by the medical community, including vague signs and symptoms, such as fatigue, depression, and abnormalities in white blood cell …

A Rare Case Of Lenalidomide Associated B Cell Lymphoblastic Leukemia, Yagnapriya Ammakola, Nitya Batra, Ashbita Pokharel, Ishmael Jaiyesimi

Conference Presentation Abstracts

Introduction Autologous stem cell transplantation followed by maintenance with Lenalidomide has improved the overall survival and progression free survival in patients with Multiple Myeloma. However, maintenance therapy with Lenalidomide can increase the risk of several hematological adverse events including secondary B cell lymphoblastic leukemia. We present a case of 61 year old female who was previously treated for multiple myeloma and on maintenance Lenalidomide developed B lymphoblastic leukemia/lymphoma. Case presentation A 61 year old female initially presented with left sided hip pain and lower back pain in 2018.Chest X ray revealed mass like opacity in the left upper lobe and …

Rising Use Of Immune Checkpoint Inhibitors And The Escalating Burden Of Gastrointestinal And Hepatic Toxicities: A Nine-Year Analysis, Donghyun Ko, Do Han Kim Md, Pedro Palacios Argueta Md, Wilhelm S. Basegoda Md, Jose A. Porres, Francis F. Fadi, Jana G. Hashash, Francis A. Farraye, Paul T. Kroner Md

Posters

The advent of immune checkpoint inhibitors (ICIs), including programmed death 1 (PD-1), PD-L1, and cytotoxic T-lymphocyte-associated antigen 4 (CTLA-4) inhibitors, has revolutionized cancer therapeutics, markedly improving survival in various malignancies. However, their expanding utilization has been paralleled by the emergence of immune-related adverse events (irAEs), particularly gastrointestinal (GI) and hepatic toxicities, which pose significant challenges in clinical practice. These toxicities can manifest as a spectrum of conditions, from mild transient symptoms to severe, life-threatening complications, necessitating a better understanding of their prevalence and trends. This study aims to evaluate the temporal changes in ICI use and associated GI and hepatic …

Long-Term Use Of Oral Factor X(A) Inhibitors Is Associated With Increased Rates Of Pancreatic Necrosis At 30- And 90-Days In Patients With Acute Pancreatitis: A Multinational Multicenter Analysis, Do Han Kim Md, Donghyun Ko, Pedro Palacios Argueta Md, Wilhelm S. Basegoda Md, Jose A. Porres, Frank J. Lukens Md, Paul T. Kroner Md

Posters

The aim of this study is to explore the association between use of long-term anticoagulation with FXaI and the odds of pancreatic necrosis in patients with AP using a large multinational database.

Evolocumab Is Associated With Lower Mortality When Compared To Statin In Masld/Mash Patients: A Multinational Analysis, Donghyun Ko, Do Han Kim Md, Wilhelm S. Basegoda Md, Jose A. Porres, Cristina H. Makarian, Luis M. Nieto Md, Pedro Palacios Argueta Md, Paul T. Kroner Md, Frank J. Lukens Md

Posters

Evolocumab is typically used as an add-on or replacement therapy for patients with dyslipidemia who are already on the maximum tolerated dose of statins or are intolerant to statins. While statins are effective in lowering lipid levels and have been shown to improve liver-related outcomes and reduce major adverse cardiovascular events (MACE), statin intolerance is increasingly encountered with their widespread use. The aim of our study is to compare the benefits of using evolocumab as monotherapy versus statins as monotherapy in patients with metabolic-dysfunction-associated steatotic liver disease (MASLD) and metabolic-dysfunction-associated steatohepatitis (MASH).

A Ticking Time Bomb: An Overview Of A Case Report Of Neutropenic Fever Secondary To Tick-Borne Illness, Yasemin Galiboglu, Danielle Thor, Joann Ha, Kristine Wong, Cindy Hou

Rowan-Virtua Research Day

The advent of immunomodulatory therapies and their ever-expanding number of treatment indications necessitates the understanding of their associated complications. Neutropenic fever serves as an example of these complications often encountered in clinical practice. Although neutropenic fever can result from virtually any pathogen, episodes of the syndrome secondary to tick-borne illness remain relatively undocumented in the scientific literature. In the case presented, a 77-year-old female with a pertinent past medical history of smoldering IgG multiple myeloma on active immunosuppressive therapy presented with a first-time episode of neutropenic fever likely secondary to tick-borne illness. Through this overview of a broader report, attention …

Increasing Incentive Spirometry Use In Patients With Sickle Cell Disease Admitted To Cmh, Juan Nario Ramirez, Alexandra Prosser-Dombrowski, Thomas R. Cochran, Joel Thompson

Posters

Acute chest syndrome is one of the leading causes of morbidity and mortality in patients with sickle cell disease. The National Heart, Lung, and Blood Institute (NHLBI) recommends that patients with sickle cell disease perform ten breaths of incentive spirometry (IS) every two hours while awake to prevent development of acute chest syndrome. By not meeting these guidelines, patients are more likely to develop this potentially life-threatening acute complication. To increase the use of IS in patients with sickle cell disease admitted to the hospital for vaso-occlusive pain. Patients were identified and IS occurrences were measured via review of electronic …

Prospective Clinical Trials Of Venetoclax And Hypomethylating Agents For Relapsed Bpdcn, Naveen Pemmaraju, Luan Hai Phan, Geoffrey Fell, Marlise R Luskin, Mahesh Swaminathan, Sherry Pierce, Courtney Dinardo, Abhishek Maiti, Marina Konopleva, Andrew A Lane

Faculty, Staff and Student Publications

No abstract provided.

Clonal Dynamics And Somatic Evolution Of Haematopoiesis In Mouse, Chiraag D Kapadia, Nicholas Williams, Kevin J Dawson, Caroline Watson, Matthew J Yousefzadeh, Duy Le, Kudzai Nyamondo, Sreeya Kodavali, Alex Cagan, Sarah Waldvogel, Xiaoyan Zhang, Josephine De La Fuente, Daniel Leongamornlert, Emily Mitchell, Marcus A Florez, Krzysztof Sosnowski, Rogelio Aguilar, Alejandra Martell, Anna Guzman, David Harrison, Laura J Niedernhofer, Katherine Y King, Peter J Campbell, Jamie Blundell, Margaret A Goodell, Jyoti Nangalia

Center on Aging Staff Publications

Haematopoietic stem cells maintain blood production throughout life1. Although extensively characterized using the laboratory mouse, little is known about clonal selection and population dynamics of the haematopoietic stem cell pool during murine ageing. We isolated stem cells and progenitors from young and old mice, identifying 221,890 somatic mutations genome-wide in 1,845 single-cell-derived colonies. Mouse stem cells and progenitors accrue approximately 45 somatic mutations per year, a rate only approximately threefold greater than human progenitors despite the vastly different organismal sizes and lifespans. Phylogenetic patterns show that stem and multipotent progenitor cell pools are established during embryogenesis, after which …

Myth Of The Pentad: Approaching Thrombotic Thrombocytopenic Purpura, Nidhi Patel, Md, Meera Jain, Natasha Edwin, Md

Providence Pharmacy PGY2 Program at Providence Medical Group 2025

Introduction: •Thrombotic Thrombocytopenic Purpura (TTP) is a thrombotic microangiopathy that leads to microthrombi formation. •Immune thrombocytopenic purpura (ITP) is an acquired autoimmune disorder characterized by a low platelet count due to both increased platelet destruction and impaired platelet production. •Though both involving thrombocytopenia, prompt recognition of TTP is critical, as it has a high mortality rate if left untreated Case Presentation: •42-year-old female with rheumatoid arthritis (RA) who was started on tocilizumab three months prior to presentation •Platelet counts dropped to 30,000 (Figure 1). Her rheumatologist presumed that it was ITP and treated her with steroids •Presented to outside emergency …

Kras Mutation Detection By Liquid Biopsy For Pancreatic Ductal Adenocarcinoma, Mahmoud Yousef, Abdelrahman Yousef, Mark W Hurd, Ashwathy Pillai, Saikat Chowdhury, Rebecca Snyder, Mark Knafl, Ryan L Lewis, Paul M Roy, Mohammad Fanaeian, Sali Albarouki, Luca F Castelnovo, Jennifer Peterson, Brandon G Smaglo, Robert A Wolff, Shubham Pant, Jason Willis, Ryan Huey, Michael Overman, Ching-Wei Tzeng, Michael P Kim, Naruhiko Ikoma, Jess E Maxwell, Matthew H G Katz, Huamin Wang, Anirban Maitra, Eugene Koay, Ethan B Ludmir, Anthony Chen, Camila Lopez, Haoqiang Ying, John Paul Shen, Dan Zhao

Faculty, Staff and Student Publications

The clinical utility of liquid biopsy (LB) for pancreatic ductal adenocarcinoma (PDAC) remain understudied. Our single-institution cohort of 311 PDAC patients with non-tumor tissues informed LB found 81.2% positivity (N = 186) in metastatic cases and in 52.4% (N = 43) of localized disease. KRAS mutations were detected in 64.6% (N = 148) of metastatic cases and 16% (N = 13) for localized disease. Positive LB, especially KRAS mutation detection, is associated with worse overall survival (OS) in metastatic PDAC (median 14.5 vs. 31.3 months, HR = 2.7, 95%CI = 1.7-4.3, P <  0.0001). The positive concordance rates of KRAS and TP53 mutations were 63% and 68% in metastatic disease but only 7% (KRAS) and 33% (TP53) in localized disease, respectively. Among the 41 patients who underwent serial liquid biopsy testing, 25% tested positive after an initial negative result. LB detects therapeutically targetable mutations in 58.5% of PDAC patients and is associated with OS.

Acute Pain Treatment In Patients With Sickle Cell Disease Transitioned To Buprenorphine: Evidence Of Equivalent Pain Relief, Christopher P. Carroll, Elizabeth J. Prince, Ashley P. Lauriello, Lydia H. Pecker, Sophie M. Lanzkron

Cardeza Foundation for Hematologic Research

Objectives Buprenorphine is a novel opioid analgesic with complex receptor pharmacology. It is safer than high dose chronic treatment with full agonist opioids. However, buprenorphine can inhibit conventional opioids, complicating acute pain treatment. Methods We compare acute pain treatment outcomes in 55 sickle cell disease patients in the year before and after transition to buprenorphine. Infusion center visits, ED visits, and hospital admissions were compared before and after transition, and pain outcomes were compared using linear mixed effects models with patients entered as random effects to account for differential contribution to the outcomes by visit rates. Results Acute care visits …

Tattoos As A Risk Factor For Malignant Lymphoma, Michael Escobar, Hajirah Farah, Anna Zhao, Hafsah Umerani, Rubab Imtiaz

Clinical Research in Practice: The Journal of Team Hippocrates

A clinical decision report using:

Nielsen C, Jerkeman M, Jöud AS. Tattoos as a risk factor for malignant lymphoma: a population-based case-control study. EClinicalMedicine. 2024;72:102649. Published 2024 May 21. https://doi.org/10.1016/j.eclinm.2024.102649

for a teenage patient contemplating getting a tattoo.

Phase 2 Trial Of Ibrutinib And Nivolumab In Patients With Relapsed Cns Lymphomas, Dai Chihara, Raphael E Steiner, Ranjit Nair, Lei Feng, Sairah Ahmed, Paolo Strati, Luis Malpica, Donna P Griffith, Shivon A Mathew, Wirt Montinez, Gita Masand, Felipe Samaniego, Maria A Rodriguez, Fredrick B Hagemeister, Luis E Fayad, Swaminathan P Iyer, Loretta J Nastoupil, Sattva S Neelapu, Christopher R Flowers, Jason R Westin

Faculty, Staff and Student Publications

Treatment options are limited for both relapsed/refractory primary and secondary central nervous system (CNS) lymphoma and the prognosis remains poor. Previous studies have shown the activity of Bruton tyrosine kinase inhibitors and programmed death-1-targeted therapies in CNS lymphoma, and studies suggested potential synergy. Therefore, we conducted a phase 2 trial that combined ibrutinib with nivolumab for patients with relapsed/refractory CNS lymphoma. Patients received 560 mg oral ibrutinib daily with 240 mg IV nivolumab every 14 days (28 days per cycle). Patients who had partial or complete response after 6 cycles of treatment could continue therapy for up to 2 years …

The Development Of Consensus Recommendation To Improve Practice Harmonization For Sickle Cell Disease Through The National Alliance Of Sickle Cell Centers, Julie Kanter, Melissa Frei-Jones, Deepa Manwani, Marsha Treadwell, Mohan Madisetti, Robin E. Miller, Seethal A. Jacob, Sana Saif-Ur-Rehman, Andrew O'Brien, Sophie Lanzkron

Cardeza Foundation for Hematologic Research

Introduction SCD remains the most common inherited blood disorder. Due to a lack of strong evidence, the management of sickle cell is often anecdotal, local to individual centers, states, and countries. Even in areas of practice with high-level data to improve practice, implementation has not been consistent. The historical lack of an agreed-upon national registry has compounded this problem and resulted in a lack of comparative effectiveness data to identify areas of deficiency and improve practice. These barriers have resulted in centers developing local policies and procedures, often with limited communication with other centers, resulting in disparate and inconsistent care. …

Case Report: Myeloid Neoplasms With The T(3;12)(Q26.2;P13.1)/ Mecom-Etv6 Translocation: Report Of Two New Cases And Review Of The Literature, Soumya Mikkilineni, Juan Pablo Pineda-Reyes, Lindsay Wilde, Andres Ferber, Zi-Xuan Wang, Stephen Peiper, Guldeep Uppal, Md, Jerald Gong, Jinglan Liu

Department of Pathology, Anatomy, and Cell Biology Faculty Papers

The MECOM (MDS1 and EVI1 complex locus) gene, located at 3q26.2, encodes an oncogenic transcription factor implicated in multiple signaling pathways. Rearrangements involving MECOM/3q26.2, including inversions, translocations, insertions and cryptic chromosomal changes, are observed in myeloid neoplasms and are associated with high-risk disease features and poor clinical outcomes. The translocation t(3;12)(q26.2;p13.1) is a rare genetic event, resulting in a fusion of the MECOM gene at 3q26.2 with the ETV6 gene at 12p13.1. To date, only 78 cases of hematologic neoplasms harboring t(3;12) have been reported in the English literature, primarily as case reports or case series. T(3;12) has …

Ven In Combination With 10-Day Dec In Newly Diagnosed Elderly Or Relapsed/Refractory Acute Myeloid Leukemia, And High-Risk Myelodysplastic Syndrome: Long Term Follow-Up Of A Phase 2 Trial, Mahesh Swaminathan, Courtney D Dinardo, Abhishek Maiti, Naveen Pemmaraju, Maro Ohanian, Navel G Daver, Guillermo Garcia-Manero, Ghayas C Issa, Gautam Borthakur, Farhad Ravandi, Guillermo Montalban-Bravo, Tapan M Kadia, Yesid Alvarado, Elias J Jabbour, Nicholas J Short, William G Wierda, Nitin Jain, Steven M Kornblau, Lucia Masarova, Sherry A Pierce, Wei Qiao, Jing Ning, Hagop Kantarjian, Marina Y Konopleva

Faculty, Staff and Student Publications

No abstract provided.

Immunotherapy Targeting A Leader Sequence Cathepsin G-Derived Peptide, Chunhua Shi, Ze Tian, Jun Yan, Mao Zhang, Pariya Sukhumalchandra, Edward Chang, Guojun Yang, Junping You, Meng Cui, Qing Shi, Celine Kerros, Anne Philips, Na Qiao, Hiroki Torikai, Sathvik Patchametla, Anna Sergeeva, Lisa St John, Helen He, Dmitri Wiederschain, Benjamin H Lee, Geraldine L C Paulus, Dongxing Zha, Jeffrey Molldrem, Gheath Alatrash

Faculty, Staff and Student Publications

Myeloid azurophil granules provide a rich source of intracellular leukemia antigens. Cathepsin G (CG) is a serine protease that has higher expression in acute myeloid leukemia (AML) blasts in comparison to normal myeloid progenitors. Based on the unique biology of HLA-A*0201 (HLA-A2), in which presentation of leader sequence (LS)-derived peptides is favored, we focused on the LS-CG-derived peptide CG1 (FLLPTGAEA). We previously detected CG1/HLA-A2 complexes on the surface of primary HLA-A2+ AML blasts and cell lines, and immunity targeting CG1/HLA-A2 in leukemia patients. T cell receptor (TCR)-mimic (m) antibodies are immunotherapeutic antibodies that target peptide-HLA (pHLA) complexes. Here we report …

Semi-Parametric Testing For Ordinal Treatment Effects In Time-To-Event Data Via Dynamic Dirichlet Process Mixtures Of The Inverse-Gaussian Distribution., Jonathan A Race, Amy S Ruppert, Yvonne Efebera, Michael L Pennell

Ambulatory and Primary Care Articles

Time-to-event data often violate the proportional hazards assumption under which the log-rank test is optimal. Such violations are especially common in the sphere of biological and medical data where heterogeneity due to unmeasured covariates or time varying effects are common. A variety of parametric survival models have been proposed in the literature which make more appropriate assumptions on the hazard function, at least for certain applications. One such model is derived from the first hitting time paradigm which assumes that a subject's event time is determined by a latent stochastic process reaching a threshold value. Several random effects specifications of …

Long Term Results Of Venetoclax Combined With Flag-Ida Induction And Consolidation For Newly Diagnosed And Relapsed Or Refractory Acute Myeloid Leukemia, Courtney D Dinardo, Wei-Ying Jen, Koichi Takahashi, Tapan M Kadia, Sanam Loghavi, Naval G Daver, Lianchun Xiao, Patrick K Reville, Ghayas C Issa, Nicholas J Short, Koji Sasaki, Sa A Wang, Jillian K Mullin, Sherry Pierce, Corey Bradley, Gautam Borthakur, Abhishek Maiti, Yesid Alvarado, Naveen Pemmaraju, Alessandra Ferrajoli, Mahesh Swaminathan, Maro Ohanian, Hussein A Abbas, Danielle Hammond, Jan Burger, Fadi Haddad, Guillermo Montalban-Bravo, Kelly Chien, Lucia Masarova, Musa Yilmaz, Nitin Jain, Michael Andreeff, Guillermo Garcia-Manero, Steven Kornblau, Farhad Ravandi, Elias Jabbour, Marina Y Konopleva, Hagop M Kantarjian

Faculty, Staff and Student Publications

Intensive chemotherapy remains the standard for newly diagnosed (ND) acute myeloid leukemia (AML); however, relapse risk remains high. Additionally, most patients with relapsed/refractory (RR) AML have poor outcomes. We report the long-term experience of 138 patients, 77 ND and 61 RR, treated with FLAG-IDA in combination with venetoclax. In the ND cohort, the overall response rate (ORR) was 97%, with a composite complete remission (CRc) rate of 95% and undetectable measurable residual disease (MRD) status by flow cytometry in 90%. The 3-year OS and EFS rates were 66 and 64%, respectively. Outcomes were similar across European LeukemiaNet (ELN) 2022 risk …

Oral Decitabine Cedazuridine With And Without Venetoclax In Higher-Risk Myelodysplastic Syndromes Or Chronic Myelomonocytic Leukemia: A Propensity Score-Matched Study, Alex Bataller, Koji Sasaki, Samuel Urrutia, Guillermo Montalban-Bravo, Alexandre Bazinet, Kelly Chien, Danielle Hammond, Ian M Bouligny, Mahesh Swaminathan, Ghayas Issa, Nicholas Short, Naval Daver, Courtney D Dinardo, Tapan Kadia, Elias Jabbour, Farhad Ravandi, Gail J Roboz, Michael Savona, Elizabeth A Griffiths, James Mccloskey, Olatoyosi Odenike, Aram Oganesian, Harold N Keer, Mohammad Azab, Hagop Kantarjian, Guillermo Garcia-Manero

Faculty, Staff and Student Publications

Hypomethylating agents (HMA) are indicated in the treatment of higher-risk myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML). The combination of hypomethylating agents with venetoclax (Ven) has demonstrated promising results in these diseases, although randomized clinical trials are needed for validation. In this retrospective study, we compared two matched cohorts of patients with MDS or CMML: one receiving oral decitabine-cedazuridine (DEC-C, n = 73) and one receiving DEC-C and Ven (DEC-C-Ven, n = 51), in three contemporary clinical trials. The aim is to determine the impact of the addition of Ven to HMA in MDS and CMML. Individuals were matched …

Oral Decitabine Cedazuridine With And Without Venetoclax In Higher-Risk Myelodysplastic Syndromes Or Chronic Myelomonocytic Leukemia: A Propensity Score-Matched Study, Alex Bataller, Koji Sasaki, Samuel Urrutia, Guillermo Montalban-Bravo, Alexandre Bazinet, Kelly Chien, Danielle Hammond, Ian M Bouligny, Mahesh Swaminathan, Ghayas Issa, Nicholas Short, Naval Daver, Courtney D Dinardo, Tapan Kadia, Elias Jabbour, Farhad Ravandi, Gail J Roboz, Michael Savona, Elizabeth A Griffiths, James Mccloskey, Olatoyosi Odenike, Aram Oganesian, Harold N Keer, Mohammad Azab, Hagop Kantarjian, Guillermo Garcia-Manero

Faculty, Staff and Student Publications

Hypomethylating agents (HMA) are indicated in the treatment of higher-risk myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML). The combination of hypomethylating agents with venetoclax (Ven) has demonstrated promising results in these diseases, although randomized clinical trials are needed for validation. In this retrospective study, we compared two matched cohorts of patients with MDS or CMML: one receiving oral decitabine-cedazuridine (DEC-C, n = 73) and one receiving DEC-C and Ven (DEC-C-Ven, n = 51), in three contemporary clinical trials. The aim is to determine the impact of the addition of Ven to HMA in MDS and CMML. Individuals were matched …

Reducing Clinical Trial Eligibility Barriers For Patients With Mds: An Icmds Position Statement, Uma Borate, Kelly Pugh, Allyson Waller, Rina Li Welkie, Ying Huang, Jan Philipp Bewersdorf, Maximilian Stahl, Amy E Dezern, Uwe Platzbecker, Mikkael A Sekeres, Andrew H Wei, Rena J Buckstein, Gail J Roboz, Michael R Savona, Sanam Loghavi, Robert P Hasserjian, Pierre Fenaux, David A Sallman, Christopher S Hourigan, Matteo Giovanni Della Porta, Stephen Nimer, Richard F Little, Valeria Santini, Fabio Efficace, Justin Taylor, Guillermo Garcia-Manero, Olatoyosi Odenike, Tae Kon Kim, Stephanie Halene, Rami S Komrokji, Elizabeth A Griffiths, Peter L Greenberg, Mina L Xu, Zhuoer Xie, Rafael Bejar, Guillermo F Sanz, Mrinal M Patnaik, Maria Figueroa, Hetty E Carraway, Omar Abdel-Wahab, Daniel Starczynowski, Eric Padron, Jacqueline Boultwood, Steven Gore, Naval G Daver, Jane E Churpek, Ravindra Majeti, John M Bennett, Alan F List, Andrew M Brunner, Amer M Zeidan

Faculty, Staff and Student Publications

Excessively restrictive inclusion and exclusion criteria in clinical trials are one of many barriers to clinical trial enrollment for patients with myelodysplastic syndromes/neoplasms (MDSs). Many organizations are developing efforts to increase clinical trial eligibility; yet, several recent publications focused on patients with MDS suggest that many patients with this disease may be excluded from clinical trials unnecessarily. Clinical trial eligibility should reflect the phase of the study and risks of the agent being studied. Phase 3 trials should be less restrictive than early-phase trials to represent the real-world population as closely as possible. We hypothesize that many clinical trials, particularly …

Unlocking Prognostic Potential: Biomarker Predictors Of Admission And Length Of Stay In Pediatric Sickle Cell Vaso-Occlusive Pain Crisis, Jeffrey Hernandez, Abhishek Dutta, Jacob Lowry, Bruce Bernstein, Luis Gamboa, Nataly Apollonsky

Tower Health Research Day

No abstract provided.

Evolution Of Btk Inhibitors, Dena Mathew

Lynchburg Journal of Medical Science

The purpose of this clinical review evaluates the evolution of bruton’s tyrosine kinase (BTK) inhibitors in becoming the standard of care in treating chronic lymphocytic leukemia (CLL). The first-generation BTK inhibitor ibrutinib has demonstrated superior efficacy over traditional chemotherapy in several randomized clinical trials in terms of progression free survival (PFS). However, due to cardiovascular toxicities of atrial fibrillation (afib), hypertension (HTN), and bleeding, have led to drug discontinuation. Second-generation BTK inhibitors, acalabrutinib and zanubrutinib have demonstrated reduced rates in cardiovascular toxicities due to improved BTK receptor selectivity, as seen in three head-to-head ibrutinib clinical trials. The emergence of BTK …

Superior Preclinical Efficacy Of Co-Treatment With Brg1/Brm And Flt3 Inhibitor Against Aml Cells With Flt3 Mutations, Warren Fiskus, Christopher P Mill, Jessica Piel, Mike Collins, Murphy Hentemann, Branko Cuglievan, Christine E Birdwell, Kaberi Das, Hanxi Hou, John A Davis, Antrix Jain, Anna Malovannaya, Tapan M Kadia, Naval Daver, Koji Sasaki, Koichi Takahashi, Danielle Hammond, Patrick K Reville, Lauren B Flores, Sanam Loghavi, Xiaoping Su, Courtney D Dinardo, Kapil N Bhalla

Faculty, Staff and Student Publications

Although treatment with standard frontline therapies, including a FLT3 inhibitor (FLT3i) reduces AML burden and achieves clinical remissions, most patients with AML with FLT3 mutation relapse due to therapy-resistant stem/progenitor cells. The core ATPases, BRG1 (SMARCA4) and BRM (SMARCA2) of the canonical (c) BAF (BRG1/BRM-associated factor) complex is a dependency in AML cells, including those harboring FLT3 mutations. We have previously reported that treatment with FHD-286, a BRG1/BRM ATPases inhibitor, induces differentiation and loss of viability of AML stem/progenitor cells. Findings of present studies demonstrate that treatment with FHD-286 induces lethality in AML cells, regardless of sensitivity or resistance to …