Digital Commons Network^™

Use Of An Electronic Medical Record Tool To Ensure Tertiary Survey Compliance In Pediatric Trauma Patients, Ryan T. Davis, Erica Surman, Begum Akay, Nathan Novotny, Anthony Stallion, Pavan Brahmamdam

Conference Presentation Abstracts

Introduction:
The purpose of the tertiary survey is to ensure that traumatic injuries that may be missed during the primary and secondary survey are identified and treated accordingly. After a review, our trauma program identified a quality gap where tertiary surveys were not being documented. The purpose of this study is to evaluate whether the use of an electronic medical record tool, in conjunction with a reporting process, improved documentation of the tertiary survey in pediatric trauma patients.

Methods:
A review of the pediatric trauma database was performed. Patients < 16 years old who were admitted after level 1 or 2 activations or trauma evaluations were included. The study period was between January 2023 and January 2024. An Epicâ„¢ SmartPhrase was developed with input from the pediatric surgery team, pediatric trauma nurse manager, and the general surgery residents. The residents and pediatric surgery attendings were educated and the smartphrase was implanted July 2023. Reviews were made on a month-to-month basis and if a patient did not have a tertiary survey documented, emails to the trauma attending and residents of record were sent, notifying them of missed documentation of a tertiary survey.

Results:
Tertiary survey documentation compliance in the six months prior to …

Lymphoma Imaging In Hiv And Non-Hiv Patients: A Retrospective Cross-Sectional Study With Clinical And Pathological Correlation, Poonamjeet Loyal, Edward Chege, Jasmit Shah, Anne Mwirigi, Samuel Gitau

Imaging & Diagnostic Radiology, East Africa

Background: Patients with Human Immunodeficiency Virus (HIV)have an atypical imaging pattern of lymphoma. There is paucity of literature on differences in tumor volume or burden of disease amongst HIV positive patients compared with HIV negative patients and how this correlates with clinicopathological parameters of aggressiveness and prognosis.

Methods: This was a retrospective cross-sectional study of patients with non-Hodgkin lymphoma which were categorized into HIV positive and HIV negative. The tumor burden, disease sites, international prognostic score and Ki-67 index were recorded. Continuous variables were analyzed using the Kruskal Wallis test and categorical variables with Fisher’s Exact test.

Results …

Not All That Hurts Is Malignant: A Painful Case Of Focal Nodular Hyperplasia, Zamanali Khakhar, Abdullahi Abdigafar Yunis, Dayib Buthul, Khadija Warfa, Soraiya Manji, Rajiv Patel, Sayed Ali Raza Bukhari

Imaging & Diagnostic Radiology, East Africa

Focal nodular hyperplasia (FNH) is a benign hepatic lesion most often detected incidentally during imaging studies. While typically asymptomatic, a small subset of patients may present with abdominal pain or discomfort. Clinically and radiologically, FNH can mimic malignant liver tumors, occasionally resulting in unnecessary invasive procedures. This case report describes a 45-year-old woman with symptomatic FNH who was placed on routine imaging surveillance and managed conservatively. This case underscores the importance of accurately distinguishing this benign condition from hepatic malignancies. It further highlights the diagnostic challenges associated with achieving a definitive diagnosis.

A Rare Case Of Ogilvie’S Syndrome In A Pediatric Patient With A Complex Medical History, Kirby Lampe, Victoria Sarata, Aileen Har

Posters

Acute colonic pseudo-obstruction (ACPO) or Ogilvie’s syndrome is an acute dilatation of the small or large bowel without an obstructive lesion. Symptoms include abdominal distension, nausea and vomiting, constipation and occasional dyspnea. The disorder most commonly involves the cecum and right colon and is most frequently seen in male patients above the age of 60 (1). It is rare for Ogilvie’s to occur in the pediatric population though some infrequent instances have been reported. Here, we report a case of a patient with significant abdominal distension and gaseous distension seen on x-ray, which was diagnosed and treated as Ogilvie’s syndrome. …

A Case Report: Ansa Pancreatica As A Cause Of Recurrent Pancreatitis, Chase Branstetter, Elizaveta Khenner, Victoria Sarata, Nadia Ibrahimi

Posters

No abstract provided.

Challenges And Opportunities With Providing Genetic Testing And Counseling For Mucopolysaccharidosis Type Ii In Kenya, Lucy Mungai, Charles Njeru, Allan Njoroge, Michuki Maina, Syokau Ilovi, Dalton Wamalwa, Ruth Nduati, Beatrice Odongkara, Danny Miller

Pathology, East Africa

Background Limited or absent genetic counseling and testing resources in low- and medium-income countries lead to missed or late diagnoses for treatable metabolic conditions with irreversible complications. In some communities, misunderstanding about the etiology of a genetic condition may lead women whose children are affected to be viewed as a bad omen and become stigmatized or ostracized from their community. Mucopolysaccharidosis type II (MPS II), or Hunter syndrome, is a lysosomal storage disorder in which deficiency or inactivity of the enzyme iduronate-2-sulfatase leads to accumulation of glycosaminoglycans throughout the body. The diagnosis can be made through clinical assessment, enzyme activity …

Bleeding (Life-Threatening): Children’S Mercy Kansas, Children's Mercy Kansas City

Clinical Pathways

No abstract provided.

Hallucinations, Children's Mercy Kansas City

Clinical Pathways

No abstract provided.

Human Sex Trafficking, Children's Mercy Kansas City

Clinical Pathways

No abstract provided.

Mortality Among Transgender Persons: A Taiwan Matched-Population And Sibling-Comparison Cohort Study, Chih-Wei Hsu, Yang-Chieh Brian Chen, Liang-Jen Wang, Mu-Hong Chen, Yao-Hsu Yang, Chih-Sung Liang, Po-Yen Chen, Edward Chia-Cheng Lai

Children’s Nutrition Research Center Staff Publications

Background: Transgender persons have been reported to experience excess mortality, but evidence is dominated by Western cohorts and seldom addresses familial confounding.

Methods: We conducted a nationwide, retrospective cohort study using two prespecified comparison designs: matched population controls (1:4, matched on legal sex and birth date) and within-family cisgender sibling comparisons. Transgender persons were identified by ≥2 psychiatrist-recorded gender identity disorder diagnoses during 2001-2021; cohort entry required age ≥6 years. Participants were followed until death or December 31, 2022. We compared all-cause, external-cause (suicide and accidents), and internal-cause mortality using Cox regression adjusted for sociodemographics and medical comorbidity.

Findings: Among …

Concerns Regarding The Standard Deviation Of Individual Responses For Assessing Treatment Response Heterogeneity, Aaron R Caldwell, David B Allison, Andrew W Brown, Gary L Gadbury, Thirupathi Reddy Mokalla, R Drew Sayer, Andrew D Vigotsky

Children’s Nutrition Research Center Staff Publications

The estimation of treatment response heterogeneity (TRH) is increasingly important as medicine moves toward personalized approaches. While various statistical methods have been proposed to quantify TRH in parallel-group trials, the standard deviation of individual responses (SDIR) has gained prominence within physiological research. This method is intended to quantify individual response variation by comparing standard deviations of change scores between intervention and control groups. We acknowledge that SDIR represents an improvement over many other flawed approaches that often involve responder counting. However, SDIR has critical limitations: 1) it cannot overcome the fundamental problem of causal inference because the correlation between potential …

Uptake Of Covid-19 Vaccination And Associated Factors Among Patients Attending Oncology Services At The Ocean Road Cancer Institute In Dar Es Salaam, Tanzania: Insights From Mixed Design Methods’ Trajectory, David Makoko, Anthony Kapesa, Micheal Mugerwa, Mamsau Ngoma, Mark Mseti, Mary Lyimo, Lucy Kisaka, Ntuli Kapologwe, Jeremiah Seni

School of Nursing & Midwifery, East Africa

Background

COVID-19 vaccination campaigns have reduced disease severity and fatalities around the globe. Global data shows that 67.7% of the general population are vaccinated, and Tanzania has reported appealing findings of over 70% coverage among individuals over 18 years of age. However, a group-specific assessment of vaccination coverage is needed in sub-Saharan Africa. This study aimed at assessing the COVID-19 vaccination uptake and associated factors among cancer patients owing to their vulnerability.

Methods

A cross-sectional analytical study design was conducted in September 2022 at Ocean Road Cancer Institute (ORCI) in Dar es Salaam, Tanzania. A multistage sampling approach was employed …

Detection Of Left Ventricular Thrombus In Newly Diagnosed Heart Failure Patients: Diagnostic Accuracy Of 2d Transthoracic Echocardiography Compared With Cardiac Mri In A Kenyan Cohort, Barbara Karau, Nadeem Kassam, Mohamed Varwani, Jeilan Mohamed, Kevin Ombati, James Orwa, Mzee Ngunga

Internal Medicine, East Africa

Background Left ventricular thrombus (LVT) is a recognized complication of severe left ventricular (LV) systolic dysfunction. While cardiac magnetic resonance (CMR) Imaging is considered the gold standard due to its high sensitivity and specificity for detecting LVT, its availability remains limited in Sub-Saharan Africa. In contrast, 2D transthoracic echocardiography (TTE) is more accessible and commonly used in clinical practice. This study aimed to determine the incidence of LVT in patients with a left ventricular ejection fraction (LVEF) of less than 35% from any etiology, and to compare the diagnostic accuracy of TTE against CMR for its detection.

Methods This prospective, …

Implementation And Evaluation Of Infection Prevention And Control Training In Sub-Saharan Africa, David Odada, James Ndai, Ruth Thuku, Philip Adebayo

Internal Medicine, East Africa

No abstract provided.

Bridging The Gap: An Educational Intervention For The Early Introduction Of Peanut Protein In Pediatric Care, Rachel Gilson Dnp, Rnc-Ob, C-Onqs, Fnp-C, Parkview Health

Nursing Publications, Posters, & Presentations

Presented at the 6th Annual Parkview Nursing Research and Evidence-Based Practice Symposium. Theme: “From Paper to Practice: The Art of Implementation Science,” at the Parkview Mirro Center for Research and Innovation.

The primary aim of this Doctor of Nursing Practice (DNP) project was to improve provider adherence to the NIAID guidelines for the early introduction of peanut protein in infants under one year of age. The project intended to address identified knowledge and practice gaps through a targeted educational intervention at Fort Wayne Pediatrics (FWP).

Use Of Robotic And Laparoscopic Surgery For Right Colectomies In Rural Areas: Are The Advantages Over Open Surgery Comparable To Those Reported In Urban Centers? An Analysis Using The Healthcare Cost And Utilization Project (Hcup) Michigan Inpatient Sample, Ryan T. Davis, Ibrahim B. Baida, Jacob A. Applegarth, Nathan M. Novotny

Conference Presentation Abstracts

Introduction: Surgical robotics have become more accessible in rural healthcare centers across Michigan, driving a growing demand for clinical outcomes data in these areas. National studies have shown that robotic colectomies reduce postoperative complications compared to open procedures, but data specific to rural settings remain limited. This study aims to compare postoperative complications following open, laparoscopic, and robotic right colectomies in rural Michigan.
Methods: The Healthcare Cost and Utilization Project (HCUP) Michigan inpatient database (2016-2018) was used to analyze postoperative complications after right colectomies in urban and rural hospitals. Complications analyzed included: prolonged ileus, pneumonia, surgical site infection, septicemia, hemorrhage/hematoma, …

Redefining Central Nervous System Multiple Myeloma: From Rare Phenomenon To Emerging Entity, Ian Landry, Adam F. Binder, Marc Yorker, Sarah Ramirez

Department of Medical Oncology Faculty Papers

BACKGROUND: Central nervous system involvement in multiple myeloma (CNS-MM) is considered a rare but devastating manifestation of extra-medullary disease (EMD) associated with aggressive biology and dismal outcomes. Treatment advances over the past few decades have improved survival in patients with MM, however, it has not translated into a similar survival benefit for those with CNS-MM. Early recognition and targeted management of CNS-MM remain major clinical challenges.

OBJECTIVE: This literature review summarizes the current literature on epidemiology, risk factors, clinical features, diagnostic modalities, and therapeutic approaches to CNS-MM. It focuses on emerging biomarkers and new treatment options that may affect the …

Proteolysis-Targeting Chimeras In Cancer Therapy: Targeted Protein Degradation For Next-Generation Treatment, Yamile Abuchard, Mariana Barragan, Ricardo Pequeno Bracho, Salique H. Shaham, Debasish Bandyopadhyay, Elias George, Diane Nguyen, Manish Tripathi

School of Medicine Publications

Proteolysis-targeting chimeras (PROTACs) have the potential to revolutionize cancer treatment by specifically targeting and degrading oncogenic proteins. Using the ubiquitin-proteasome system, PROTACs allow the selective degradation of disease-causing proteins, including those traditionally deemed “undruggable” by conventional small-molecule inhibitors. By catalytically eliminating rather than inhibiting proteins, PROTACs provide sustained target suppression with lower doses and reduced toxicity. Their bifunctional design linking a protein of interest to an E3 ligase drives targeted ubiquitination and subsequent proteasomal degradation. Recent progress demonstrates promise in treating solid and hematologic malignancies, with several candidates advancing to clinical trials. This review provides a comprehensive overview of developing …

Use Of Prophylaxis For Deep Venous Thrombosis And Pulmonary Embolism In Isolated Foot And Ankle Trauma, Regina Hansen, Alexander P. Rader, Naohiro Shibuya, Daniel C. Jupiter

School of Podiatric Medicine Publications

Background: Deep venous thrombosis (DVT) and pulmonary embolism (PE) are relatively rare after foot and ankle surgery, especially after trauma. Diagnosing DVT is difficult, and even when it is discovered it does not necessarily clinically manifest on, for example, duplex ultrasound. Thus, it is often recommended that prophylaxis use be assessed on a case-by-case basis based on risk profile rather than being uniformly given or not prescribed.

Methods: We searched the National Trauma Data Bank Trauma Quality Programs Participant Use File for patients experiencing foot and ankle trauma from 2013 to 2017. Patients were identified using the International Classification of …

Ear Foreign Body, Children's Mercy Kansas City

Clinical Pathways

No abstract provided.

Single-Cell-Driven Design Of Logic-Gated Car Circuits For Enhanced Solid Tumor Immunotherapy, Sanna Madan, Tian-Gen Chang, Alexandra Harris, Huaitian Liu, Andrew Martinez, Saugato Dhruba, Binbin Wang, Padma Rajagopal, Sanju Sinha, Shahin Sayed

Pathology, East Africa

Background Chimeric antigen receptor (CAR) T-cell therapy has revolutionized cancer immunotherapy for hematological malignancies but faces critical barriers in solid tumors due to tumor antigen heterogeneity and on-target, off-tumor toxicities. Logic-gated multi-antigen targeting using Boolean operators (AND, OR, NOT) represents an emerging precision immunotherapy engineering approach to overcome these challenges.

Methods We developed LogiCAR designer, a genetic algorithm-based computational framework that systematically identifies optimal logic-gated antigen combinations (i.e., ‘circuits’) from single-cell transcriptomics data. The algorithm identifies CAR circuits consisting of 1-5 antigens from 2,758 cell surface proteins, optimizing tumor-targeting efficacy while maintaining stringent safety requirements in normal tissues. …

Who Classification Of Skin Tumours: Key Updates In The Fifth Edition, Gabrielle Goldman-Lévy, Raymond Barnhill, Boris Bastian, Werner Kempf, David Elder, Pedram Gerami, Wayne Grayson, Dmitry Kazakov, Daniela Massi, Shahin Sayed

Pathology, East Africa

The 5th edition of the World Health Organization Classification of Tumours (WCT) serves as a foundation for global diagnostic standards in tumour pathology. Similar to other volumes in this series, the Skin Tumours (Skin5) edition follows a standardized approach. This edition introduces two new chapters: ‘Tumours of the nail unit’ and ‘Metastases to skin’, along with new entities across relevant chapters. This review article provides an overview of the updates in Skin5 based on currently published evidence, with emphasis on newly introduced chapters and newly described entities that involve the skin, in particular, epidermal, melanocytic and appendageal tumours.

Microbial Signals In Primary And Metastatic Brain Tumors, Golnaz Morad, Ashish V Damania, Brenda Melendez, Bharat B Singh, Fabiana J Veguilla, Rebecca A Soto, Yasmine M Hoballah, Pranoti V Sahasrabhojane, Matthew C Wong, Mona M Ahmed, Rene N Rico, Kaitlyn N Lewis, Khalida Wani, Diana D Shamsutdinova, Rossana N Lazcano Segura, Davis R Ingram, Eric A Goethe, Abderrahman Day, Ivonne I Flores, Lauren K Mcdaniel, Manoj Chelvanambi, Sarah B Johnson, Florentia Dimitriou, Pravesh Gupta, Shivangi Oberai, M Anna Zal, Phoebe Doss, Mohamed A Jamal, Eiko Hayase, Chetna Wathoo, Lisa M Norberg, Stephanie L Jenkins, Sara Nass, Joy Gumin, Lihong Long, Jing Yang, Gina R Bradley, Mahesh Prasad Bekal, Antonio G Dono, Pavel S Pichardo-Rojas, Samuel W Andrewes, Leomar Y Ballester, Jillian S Losh, Jiyong Liang, Longfei Huo, Douglas C Nielsen, Brittany C Parker Kerrigan, Priscilla K Brastianos, Natalie Wall Fowlkes, Chia-Chi Chang, Robert R Jenq, Candelaria Gomez-Manzano, Jason T Huse, Michael A Davies, Alexander J Lazar, Krishna P Bhat, Nitin Tandon, Yoshua Esquenazi, Christine B Peterson, Vinay K Puduvalli, Frederick F Lang, Christopher D Johnston, Susan Bullman, Nadim J Ajami, Sherise D Ferguson, Jennifer A Wargo

Faculty, Staff and Student Publications

Gliomas and brain metastases are associated with poor prognosis, necessitating a deeper understanding of brain tumor biology and the development of effective therapeutic strategies. Although our group and others have demonstrated microbial presence in various tumors, recent controversies regarding cancer-type-specific intratumoral microbiota emphasize the importance of rigorous, orthogonal validation. This prospective, multi-institutional study included a total of 243 samples from 221 patients, comprising 168 glioma and brain metastases samples and 75 non-cancerous or tumor-adjacent tissues. Using stringent fluorescence in situ hybridization, immunohistochemistry and high-resolution spatial imaging, we detected intracellular bacterial 16S rRNA and lipopolysaccharides in both glioma and brain metastases …

Colorectal-Specific Radiation Dose And Chemotherapy Risk For Subsequent Colorectal Malignancies In Childhood Cancer Survivors: A Childhood Cancer Survivor Study (Ccss) Report, Constance A Owens, Ethan B Ludmir, Qi Liu, Weiyu Qiu, Aashish C Gupta, Susan A Smith, Bastien Rigaud, Kristy K Brock, James E Bates, Taylor G Meyers, Arnold C Paulino, Christine B Peterson, Stephen F Kry, Jop C Teepen, Cécile M Ronckers, Joseph P Neglia, Wendy M Leisenring, Kevin C Oeffinger, Paul C Nathan, Lucie M Turcotte, David C Hodgson, Melissa M Hudson, Leslie L Robison, Chaya S Moskowitz, Gregory T Armstrong, Tara O Henderson, Yutaka Yasui, Rebecca M Howell

Faculty, Staff and Student Publications

Purpose: Among childhood cancer survivors, we evaluated not previously explored relationships between colorectal subsequent malignant neoplasm (SMN) incidence and colorectum-specific radiation dose metrics currently used in radiation therapy (RT) planning and expanded upon previously reported chemotherapy associations.

Methods: The Childhood Cancer Survivor Study (CCSS) includes 5-year survivors of childhood cancer diagnosed between 1970 and 1999. RT was assessed as mean colorectal dose (MCD) and the percent volume (VX Gy) receiving ≥5, 10, 20, 30, and 40 Gy. Chemotherapy was assessed as cumulative doses for procarbazine and platinum agents, cyclophosphamide-equivalent doses for alkylating agents, and doxorubicin-equivalent doses for anthracyclines. Piecewise-exponential models …

Intentional Creation Of Suboptimal, Realistic Dose Distributions, Skylar S Gay, Mary P Gronberg, Raymond Mumme, Beth M Beadle, Anuja Jhingran, Tze Yee Lim, Zhiqian H Yu, Christine Chung, Meena Khan, Chelsea Pinnix, Sanjay Shete, Brent Parker, Tucker J Netherton, Carlos E Cardenas, Laurence E Court

Faculty, Staff and Student Publications

Background: Radiation oncology residents report a lack of understanding and confidence in assessing radiotherapy plan quality. A contributing factor is the environment in which plan review is taught during residency, that is, routine clinical practice, which does not provide ample time for self-guided practice in a low-stakes setting. Expertise in plan review requires diverse case presentation and many examples, which are often not achievable in smaller programs and for less common cancer types. As plan quality affects patient outcomes, it is important to address these pitfalls in the education of residents on plan review.

Purpose: To address the identified pitfalls …

Outcomes And Patterns Of Relapse Of Npm1 Mutated Acute Myeloid Leukemia Treated With Venetoclax Based Therapies, Wei-Ying Jen, Sanam Loghavi, Alexandre Bazinet, Alex Bataller, Ian Bouligny, Naval G Daver, Ghayas C Issa, Naszrin Arani, Emmanuel Almanza Huante, Abhishek Maiti, Guillermo Montalban-Bravo, Gautam Borthakur, Nicholas J Short, Sherry Pierce, Elias Jabbour, Guillermo Garcia-Manero, Farhad Ravandi, Hagop M Kantarjian, Courtney D Dinardo, Tapan M Kadia

Faculty, Staff and Student Publications

No abstract provided.

A Tour Of Leukemia Progress In 2025, Viewed Through The Md Anderson Leukemia Research Lens, Hagop M Kantarjian, Gautam Borthakur, Naval Daver, Courtney Dinardo, Guillermo Garcia-Manero, Ghayas Issa, Elias Jabbour, Nitin Jain, Tapan Kadia, Sanam Loghavi, Farhad Ravandi, Guilin Tang, Mary Alma Welch, William Wierda

Faculty, Staff and Student Publications

Advances in the prognostication, monitoring, and treatment of both the acute and chronic leukemias have led to drastically improved outcomes over the past 2 decades. With the advent of targeted therapies, including antibodies such as blinatumomab and inotuzumab and small molecule inhibitors, such as the BCR::ABL1 tyrosine kinase inhibitors, Bruton tyrosine kinase inhibitors, and venetoclax, the treatment landscape of leukemia has drastically changed, improving survival outcomes while relying less on overall chemotherapy intensity in many leukemia types. This progress has allowed the categorization of more leukemia types as favorable (i.e., chronic lymphocytic leukemia, younger acute lymphoblastic leukemia [patients younger than …

Brain-Wide Patterns Of Crf Receptor 1 Expression In The Transgenic Crfr1-Cre-2a-Tdtom Rat, Douglas S Engelke, Lierni Ugartemendia, Jonathan Tao, Shivakumar Rajamanikam, Fabricio H Do Monte, Nicholas J Justice

Faculty, Staff and Student Publications

The ability to manipulate neurons that express corticotropin-releasing factor (CRF) or its receptors has led to many new discoveries about how CRF signaling modulates stress-related behaviors in mice. However, similar advances in rats have been limited by the lack of reporter lines targeting CRF receptors, restricting access to behavioral paradigms that are more suitable for, or specific to, rats. Here, we provide an extensive neuroanatomical characterization of a recently generated CRFR1-Cre rat. We show that Cre and tdTomato are expressed in a pattern that closely aligns with previously reported CRFR1 expression patterns, as determined by in situ hybridization in rats …

Probabilistic Template Matching For Detecting Resting-State Functional Mri Language Network In Brain Tumor Patients, Jian Ming Teo, Vinodh A Kumar, Alexander M Khalaf, Kyle R Noll, Sherise D Ferguson, Chibawanye I Ene, Sujit S Prabhu, Max Wintermark, Ho-Ling Liu

Faculty, Staff and Student Publications

Background: Intersubject variation among patients with brain tumors complicates the template matching process for detecting the resting-state (rs) functional MRI (fMRI) language network when using independent component analysis (ICA).

Purpose: This study aimed to develop methods that use a probabilistic language atlas to incorporate intersubject variation in brain tumor patients into the template matching process.

Methods: This retrospective study included 79 patients with brain tumors (average age, 50 ± 15 years) who underwent presurgical task-based (tb)-fMRI and rs-fMRI at clinical 3T scanners. At varying template generation thresholds (τ), binary and probabilistic templates were obtained from the language atlas. A binary …

Tagraxofusp, A Cd123-Targeted Therapy, For Chronic Myelomonocytic Leukemia: Final Results Of A Phase 1/2 Study, Mrinal M Patnaik, Haris Ali, Eunice S Wang, Abdulraheem Yacoub, James M Foran, Vikas Gupta, Gary J Schiller, Don Ambrose Stevens, Moshe Talpaz, Sarah Wall, Terra L Lasho, Abhishek A Mangaonkar, Talha Badar, Ross Lindsay, Antonio Galleu, Ira Gupta, Naveen Pemmaraju, Guillermo Garcia-Manero

Faculty, Staff and Student Publications

Chronic myelomonocytic leukemia (CMML) is an aggressive hematologic neoplasm characterized by an expansion of CD123+ monocytes and plasmacytoid dendritic cells (pDCs). pDC bone marrow clusters in CMML have been associated with higher rates of acute myeloid leukemia transformation. We evaluated tagraxofusp, a CD123-targeted therapy, in a phase 1/2 trial for patients with CMML. There were no dose-limiting toxicities. At the recommended phase 2 dose of 12 μg/kg per day, 37 patients were treated: 15 were treatment naïve; 22 had relapsed/refractory disease (median number of previous therapies, 1 [range, 1-7]). Common nonhematologic treatment-emergent adverse events (AEs) included fatigue (49%), hypoalbuminemia (46%), …