Medical Specialties Commons^™

Phase 2 Study Of Monotherapy With Pembrolizumab For Advanced Adrenocortical Carcinoma, Brenda Chahla, Bettzy Stephen, Juhee Song, Vania Balderrama-Brondani, Feyza Yaylaci, Matthew T Campbell, Aung Naing, Mouhammed Amir Habra

Faculty, Staff and Student Publications

Introduction: Adrenocortical carcinoma (ACC) is a rare cancer with suboptimal response to chemotherapy. The role of immunotherapy in ACC management is evolving.

Methods: An investigator-initiated, open-label, phase 2 clinical trial was performed to ascertain the activity and safety of monotherapy with pembrolizumab (a humanized monoclonal anti-programmed cell death protein 1 antibody) in patients with advanced ACC. This study was part of a basket clinical trial (ClinicalTrials.gov ID: NCT02721732). Study participants were enrolled from August 15, 2016, until December 7, 2020. Pembrolizumab (200 mg) was administered intravenously every 3 weeks. All other lines of therapy, including mitotane, were stopped before …

Omacetaxine And Venetoclax In Relapsed/Refractory Acute Myeloid Leukemia Or Myelodysplastic Syndrome With Mutant Runx1, Courtney D Dinardo, Wei-Ying Jen, Guillermo Montalban-Bravo, Xuemei Wang, Sanam Loghavi, Sravanthi Lavu, Nicholas J Short, Kelly Chien, Ghayas C Issa, Naveen Pemmaraju, Musa Yilmaz, Michael Andreeff, Gautam Borthakur, Tapan M Kadia, Naval G Daver, Guillermo Garcia-Manero, Christopher P Mill, Xiaoping Su, Warren Fiskus, Kapil N Bhalla

Faculty, Staff and Student Publications

Mutations in RUNX1 (RUNX1mut) occur in 10% to 20% of patients with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) and are associated with poor outcomes to standard therapy. Omacetaxine mepesuccinate (OM), a semisynthetic analog of homoharringtonine, has been shown to be lethal to RUNX1mut AML cells in vitro through reduction of MCL1 and BCL-XL, and synergizes with venetoclax (VEN) in RUNX1mut AML models. We investigated the safety and efficacy of OM + VEN in relapsed/refractory RUNX1mut MDS/AML in a Bayesian Optimal Interval design. VEN 400 mg daily from days 1 to 14 and OM …

Cardiovascular Multimorbidity In Older Adults In The United States By Race And Sex, Michael D Green, Robert J Mentz, Stephen J Greene, Bradley G Hammill, Utibe R Essien, Ying Xian, Erin D Michos, Roland J Thorpe, Matthew E Dupre, Brian Mac Grory, Chi-Tsun Chiu, Emily C O'Brien, Jay B Lusk

Faculty, Staff and Student Publications

Background: It is essential to understand the prevalence of cardiovascular multimorbidity and to recognize disparities by race and sex to promote health equity.

Objectives: The objectives of the study are to investigate disparities in the development and progression of cardiovascular multimorbidity among older adults in the United States and estimate relative life expectancies among patients with cardiovascular multimorbidity.

Methods: This was a nationwide study of a 5% nationwide sample of fee-for-service Medicare beneficiaries aged 65 or older from 2010 to 2020. Multistate survival models were employed to estimate cardiovascular disease (CVD) progression and a microsimulation approach was used to derive …

Surgical Outcomes With Neoadjuvant Durvalumab Plus Chemotherapy Followed By Adjuvant Durvalumab In Resectable Nsclc, Tetsuya Mitsudomi, John V Heymach, Martin Reck, Janis M Taube, Shugeng Gao, Yoshitsugu Horio, Jian You, Gaofeng Li, Dinh Van Luong, Somcharoen Saeteng, Fumihiro Tanaka, Stefan B Watzka, Laszlo Urban, Zsuzsanna Szalai, Hiroaki Akamatsu, Jin Hyoung Kang, Francisco J Orlandi, Guzel Z Mukhametshina, Andreas Pircher, Carlos Henrique Andrade Teixeira, Mike Aperghis, Gary J Doherty, Ruth Doake, Tamer M Fouad, David Harpole

Faculty, Staff and Student Publications

Introduction: In AEGEAN, perioperative durvalumab plus neoadjuvant chemotherapy, versus neoadjuvant chemotherapy alone, significantly improved event-free survival (p = 0.004) and pathologic complete response (p < 0.001; primary end points; modified intention-to-treat [mITT] population, which excluded patients with known EGFR or ALK aberrations) with a manageable safety profile in patients with resectable (R)-NSCLC. Here, we report surgical outcomes from AEGEAN.

Methods: Patients with treatment-naive R-NSCLC (stage II-IIIB [N2]) and Eastern Cooperative Oncology Group performance status 0 or 1 were randomized (1:1) to platinum-based chemotherapy plus durvalumab or placebo intravenously (every 3 wk, 4 cycles) before surgery, followed by durvalumab or placebo (every 4 wk, 12 cycles). Surgical outcomes were summarized for the mITT population using descriptive statistics.

Results: A total of 737 out of 740 mITT patients received treatment, 366 and 371 in the durvalumab and …

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 …

Correction: A Comprehensive Narrative Review Of The Challenges Surrounding Cutaneous Scc., Sarah C Beach, Austin S Cusick, Aaron S Farberg, Shannon C Trotter

Ambulatory and Primary Care Articles

No abstract provided.

The Epidemiology Of Us Upper Extremity Amputations: An Analysis Of The Global Burden Of Disease Database (1990-2019)., Ambrose Loc T Ngo, Cameron J Sabet, Gabrielle Dykhouse, Ayah Ibrahim, Taylor J Manes, Arsalaan Sayyed, Shaheryar Asad, Benjamin C Taylor

Surgery Articles

Background Upper extremity amputations (UEAs) are medically, psychologically, and functionally devastating. This study aimed to evaluate region- and sex-specific differences in unilateral and bilateral upper extremity amputations in the United States (US) from 1990 to 2019. Methods The Global Burden of Disease (GBD) database was used to analyze years lived with disability (YLDs), prevalence, and incidence rates per 100,000 people for upper extremity amputations in the US from 1990 to 2019. Data were stratified into four US Census Bureau-defined regions: Northeast, Midwest, South, and West. Differences between regions and sexes were assessed, with statistical significance defined as p< 0.05. Results Between 1990 and 2019, unilateral upper extremity amputations in the US showed a 34.51% decrease in YLDs, 34.34% in prevalence, and 37.38% in incidence. Bilateral amputations decreased by 51.32%, 48.94%, and 56.80%, respectively. Men consistently had higher YLDs, prevalence, and incidence rates for unilateral amputations (p< 0.001). Regionally, the Northeast had the highest mean YLDs, prevalence, and incidence, while the South had the lowest. Men also experienced significantly higher rates of both unilateral and bilateral amputations across all regions compared to women (p< 0.05). Conclusions From 1990 to 2019, the US experienced declines in YLDs, prevalence, and incidence of upper extremity amputations. Men had higher rates than women across all regions. The Northeast exhibited the highest rates, while the South had the lowest. These findings underscore notable gender and regional disparities in upper extremity amputation trends.

Telepsychiatry For Substance Use Disorders: Evolution From Rapid Covid-19 Pandemic Adoption To Sustainable Integration, Jack Wang

Marshall Journal of Medicine

ABSTRACT

Introduction: Prior to the COVID-19 pandemic, telepsychiatry for substance use disorder (SUD) management was generally an underutilized modality, mainly due to regulatory constraints. Through the pandemic, governmental deregulations rapidly and massively expanded their adoption, bringing out their significant potential to expand access, overcome barriers, and mitigate stigma for individuals seeking addiction care, particularly for rural and underserved populations. This narrative review consolidates the recent research on the growing adoption of telepsychiatry in the management of SUDs. It aims to explore the evidence-based benefits, identify common challenges, and underscore key considerations for its effective and equitable integration into SUD treatment …

Lyme Disease Presenting As A Dermal Hypersensitivity Reaction, William T. Snider, Ian Depew, Rebecca Hicks, Shane Cook

Marshall Journal of Medicine

Background:

Lyme disease is a well-recognized illness commonly associated with skin manifestations such as erythema migrans. However, atypical presentations can complicate diagnosis, particularly when they deviate from classic clinical and histological findings.

Methods:

We report a potential case of Lyme disease presenting as a dermal hypersensitivity reaction (DHR), with clinical findings resembling urticaria rather than urticarial vasculitis. Skin biopsy findings were reviewed, and Lyme serology testing was performed.

Results:

The biopsy did not demonstrate the characteristic superficial perivascular lymphocytic infiltrate typically seen with erythema migrans. The patient's history included prior tick exposures, but while Lyme IgM was positive, the Lyme …

Nonglomerular Hypocomplementemic Interstitial Nephritis, Tyler Hemphill, Yogesh Sanghvi

HCA Healthcare Journal of Medicine

Background

Acute interstitial nephritis (AIN) is the most common cause of acute kidney injury (AKI) with subsequent renal impairment and is often underdiagnosed. Acute interstitial nephritis presents with a decline in kidney function and is characterized by an inflammatory infiltrate in the kidney interstitium. It is most often induced by drug therapy and is sometimes followed by infections with unknown causes. In a typical presentation of AIN, there is no evidence of hypocomplementemia; low complement is associated most commonly with autoimmune etiologies. Renal biopsy serves as the most definitive test for both the diagnosis and prognosis of AIN.

Case Presentation …

Use Of Topical Hemostatic Agents In A Gynecologic Patient With Coagulopathy, Alexis O'Connell, Syeda Zainab Shah

HCA Healthcare Journal of Medicine

Introduction

The use of topical hemostatic agents has become common during surgical interventions. There is a wide variety of substances available to augment clotting, ranging from physical agents to topical thrombin combinations. Application of these agents in gynecologic surgery in patients with existing coagulopathies has not been studied.

Case Presentation

A 62-year-old woman with an inferior vena cava filter and a history of multiple deep vein thromboses presented to the clinic with pelvic pain, stress urinary incontinence, and uterovaginal prolapse. She underwent a total vaginal hysterectomy, sacral colpopexy, tension-free vaginal tape sling, and cystourethroscopy. Warfarin was cross titrated to enoxaparin …

Utilizing Femoral Metaphyseal Sleeves In Primary Total Knee Arthroplasty For Post-Traumatic Osteoarthritis: A Case Report, Zachary Sukin, Adam Dentinger, Jeremiah Earl

HCA Healthcare Journal of Medicine

Background

Total knee arthroplasty in younger patients presents a heightened risk of postoperative complications and implant failures compared to older populations, particularly in those with posttraumatic arthritis. These challenges necessitate careful consideration and specialized approaches to optimize the outcomes in this patient demographic.

Case Presentation

A 43-year-old man presented with left knee pain resulting from a traumatic lawn mower injury, causing an intraarticular distal femur, at the age of 6. The injury led to a limb-length discrepancy for which he underwent 2 limb-lengthening procedures in his early 20s. Radiographs demonstrated advanced posttraumatic osteoarthritis, and the patient elected to proceed with …

Upper Respiratory Tract Infection Leading To A New Diagnosis Of Sweet Syndrome And Monoclonal Gammopathy Of Unknown Significance, Nidhi Rawat, Meron Gebrehiwot, Jason Raw, Jeyaprakash Ramachandran

HCA Healthcare Journal of Medicine

Background

We present a rare case of Sweet syndrome with underlying monoclonal gammopathy of unknown significance (MGUS) which initially presented as upper respiratory tract infection.

Case Presentation

A 52-year-old woman presented with a complaint of sore throat for 6 days, productive cough and fever for 5 days, and red, pruritic, circular, tender rashes on face, arms and trunk for 2 days. There was a past history of similar self-limiting rashes presenting intermittently for 1.5 years. She also reported to be taking tablet ibuprofen, as required for the past 1-2 years, for cervical spondylosis. On integumentary examination, widespread, red, tender, annular …

Capacity Over Cognition: A Case Of High-Risk Disease And Homelessness, Mikalah E. Maury, Avery M. Duncan, Rebecca A. Taylor, Mary Ann Beil, Maulikkumar A. Patel

HCA Healthcare Journal of Medicine

Background

Medical decision-making in indigent patients with baseline cognitive deficits and lack of supportive resources is a difficult problem that reveals many ethical issues. Performing high-level medical procedures (eg, invasive surgery, chemotherapy, radiation therapy) on these patients without supportive resources brings into question the efficacy and necessity of treatment if it places the patient at higher risk of additional complications and mortality.

Case Presentation

In this article, we present a case of a 62-year-old indigent man with a long history of encephalopathy secondary to alcohol abuse, presenting with an aggressive head and neck cancer impacting his quality of life.

Conclusion …

Rapid Percutaneous Nephrostomy Stent Encrustation In Pregnancy: Navigating Your Stone Challenge, Victor M. Feldbaum, Roberto Morales, Shawn R. Ramsey, Michael L. Douso

HCA Healthcare Journal of Medicine

Background

Surgical management of kidney stones in pregnancy can be a serious problem for both mother and fetus.

Case Presentation

A 20-year-old woman with a second trimester pregnancy presented to the emergency room with renal calculi. She required surgical intervention and a left nephrostomy tube after consultation with urology. Five weeks later, her tube was solidified in place unbeknownst to her care team. She required several surgeries, dealt with encrustation remnants, infections, radiation exposure, persistent infection requiring antibiotic management, and, ultimately, an early delivery at 37 weeks.

Conclusion

At the time of writing, this was the first case to describe …

Rectal Fidaxomicin As Salvage Therapy In Fulminant Clostridioides Difficile Infection After Total Colectomy: A Case Report, Akankcha Alok, Mohamed Ismail, Stephanie Pino Fabara, Mohammed Al Said, Vidya Sagar Kollu

HCA Healthcare Journal of Medicine

Introduction

Clostridioides difficile infection (CDI) is a significant cause of antibiotic-associated diarrhea and colitis, ranging from mild to severe and potentially life-threatening conditions, like fulminant colitis with toxic megacolon and bowel perforation. The 2021 guidelines from the Infectious Diseases Society of America (IDSA) and Society for Healthcare Epidemiology of America (SHEA) recommend vancomycin and metronidazole for fulminant CDI (FCDI), with consideration of rectal vancomycin for cases with ileus. Fidaxomicin, though preferred for initial and recurrent CDI treatment, is not the first line for FCDI due to limited evidence. Fidaxomicin has a narrow spectrum of activity only targeting C difficile and …

Clinical Images Of Cutaneous Horns In Patients Presenting To A Family Medicine Residency Practice, Julia Fashner, Tillie Schumann

HCA Healthcare Journal of Medicine

We detail 2 cases of cutaneous horn, also referred to as cornu cutaneum, which is a hard-compacted linear outgrowth of keratin that projects above the skin. We present these images to increase awareness for all medical specialties who may see a patient with this skin lesion.

Clinical Characteristics Associated With Hospital Mortality And Length Of Stay Among Older Adults Admitted With Delirium: Retrospective Analysis, Se Won Lee, Jeff J. Kim, Cole Kretchmar, Napatkamon Ayutyanont, Mooyeon Oh-Park

HCA Healthcare Journal of Medicine

Background

Limited information exists on the characteristics of older adults admitted with delirium at risk of poor hospital outcomes. This study aims to identify risk factors present on admission that are associated with hospital mortality and length of stay (LOS) among older adults admitted with delirium.

Methods

This is a retrospective study of 5598 patients 65 years of age or older who were admitted to acute care units at 8 community hospitals with an International Statistical Classification of Diseases, Tenth Revision code of delirium.

Results

Multivariate regression analyses revealed that in-hospital mortality increase was associated with increased age, Asian race, …

Can The Combination Of White Blood Cell Count And C-Reactive Protein Rule Out Pediatric Appendicitis, A Retrospective Analysis?, Scott Gutovitz, Samantha Stoner, Jarrett Koper, Sarah Kleist

HCA Healthcare Journal of Medicine

Background

Pediatric appendicitis (PA) is the most frequent cause of pediatric emergency surgery. Standard PA scoring systems incorporate inflammatory biomarkers (white blood cell count [WBC] or C-reactive protein [CRP]); however, the individual biomarker sensitivities have not been reported beyond single-center sites. We aimed to measure the individual and combined sensitivities of WBC and CRP to rule out PA in a large multicenter hospital system.

Methods

We did a retrospective study of pediatric emergency department patients (age <18) with abdominal pain, using a deidentified electronic health database from a 175-hospital system in the United States, comparing PA versus non-PA patients. Pediatric appendicitis patients were identified by ICD-10 diagnosis codes and had advanced imaging, whereas non-PA patients had advanced imaging and were discharged home with the ICD-10 code for abdominal pain. Using matched propensity matching, we calculated receiver operator characteristics and Youden’s index to find the optimal cutpoints. Sensitivity was calculated at the optimal and traditional cutpoints for each biomarker, also combined in parallel fashion.

Results

We identified 7414 subjects (3707 PA matched to 3707 controls). The WBC in PA patients was 14.99 ± 5.00 k/mm³ versus 9.63 …

Beyond The Norm: Decoding Spontaneous Coronary Artery Dissection As An Unusual Cause Of Acute Coronary Syndrome, Holden Zimmerman, Himanshi Banker, Inder P. Kaur, Jaskaran Munjal, Vasu Gupta, Ayushi Garg, Nikita Garg, Meet Patel, Rohit Jain

HCA Healthcare Journal of Medicine

Spontaneous coronary artery dissection (SCAD) is a rare nonatherosclerotic cause of myocardial infarction among young women, particularly during pregnancy. As the name suggests, SCAD is caused by the spontaneous formation of the false lumen within the coronary artery wall, thus compromising the blood flow to the myocardium. Unlike atherosclerotic acute coronary syndromes, SCAD is not directly associated with diabetes or hypertension; moreover, underlying connective tissue defects and inflammation are considered more contributory towards its etiology. Although its pathophysiology is not well understood, multiple theories have been proposed. Clinically, SCAD has a similar presentation to acute coronary syndromes, including substernal chest …

Circulating Inflammatory Biomarkers And Endometriosis Lesion Characteristics In The Wise Consortium, Kathryn L. Terry, Amy Shafrir, Ashley Laliberte, Allison F. Vitonis, Kurtis Garbutt, Mary Depari, Christian Becker, Naoko Sasamoto, Krina T. Zondervan, Stacey A. Missmer

Health Sciences Faculty Publications

Endometriosis is a chronic inflammatory condition requiring surgical or imaging visualization for definitive diagnosis. How endometriotic lesion characteristics relate to circulating inflammatory markers remains unclear. We evaluated 11 inflammatory biomarkers, including interleukin (IL)-1β, -6, -8, -10, -16, tumor necrosis factor (TNF)-α, thymus and activation regulated chemokine (TARC), monocyte chemotactic protein (MCP)-1, -4, and Interferon gamma-induced protein (IP)-10, in 566 participants with endometriosis from the Women’s Health Study: From Adolescence to Adulthood (A2A), Endometriosis Oxford Care & Research (CaRe) study (ENDOX) and Endometriosis: Natural History, Diagnosis, and Outcome (ENDO) study to evaluate associations with endometriosis characteristics, including macrophenotype (superficial lesions only, …

Atypical Abdominal Pain: Uncovering Malignant Peritoneal Mesothelioma In Suspected Crohn's Disease: A Case Report, Catherine Loehr, Lara Boudreaux, Ann Porter Uhlhorn, Nisha Loganantharaj

School of Medicine Faculty Publications

BACKGROUND: Malignant peritoneal mesothelioma (MPM) is a rare and aggressive cancer from the serosal membranes of the abdominal cavity. In the United States, MPM accounts for about 10-15% of all mesothelioma cases, and translates into approximately 600 new cases diagnosed annually. It is typically diagnosed in Caucasian individuals, and while asbestos exposure is a well-established risk factor for pleural mesothelioma, its association with peritoneal mesothelioma remains less clear. Clinical symptoms of MPM, such as abdominal pain, bloating, and weight loss, overlap with other gastrointestinal disorders, which often leads to delayed diagnosis. This case report presents a unique instance of MPM …

Urinary Vitamin D Binding Protein And Kidney Injury Molecule-1 Are Potent Predictors Of Acute Kidney Injury After Left Ventricular Assist Device Implantation, Shiyi Li, Iván Murrieta-Álvarez, Katherine V Nordick, Zachary Gray, Camila Hochman-Mendez, Alexis E Shafii, Kenneth K Liao, Carl P Walther, Nandan K Mondal

Faculty, Staff and Students Publications

Background/Objectives: Acute kidney injury (AKI) is a frequent and serious complication following left ventricular assist device (LVAD) implantation. This study aimed to predict AKI within 90 days post-LVAD by evaluating urinary levels of vitamin D binding protein (VDBP) and kidney injury molecule-1 (KIM-1).

Methods: We prospectively enrolled 29 advanced heart failure patients undergoing LVAD implantation and categorized them into four groups based on pre-LVAD kidney function and hemodynamic stability. Early-morning urine samples were collected 24 h before and 1 week after surgery. Urinary VDBP and KIM-1 levels, normalized to creatinine, were measured.

Results: Thirteen patients developed AKI …

Safety And Efficacy Of Liver-Directed Radiotherapy After Chemoimmunotherapy For Advanced Cholangiocarcinoma: An Early Report From A Large Tertiary Cancer Center, Abdulmoiz Asif, Abdulmoid Asif, Michael K Rooney, Enoch Chang, Felicity Namayanja, Kimana Quentin, Sunyoung Lee, Ian Hu, Vincent Bernard, Prajnan Das, Emma B Holiday, Sonal Noticewala, Grace L Smith, Albert C Koong, Ethan B Ludmir, Milind Javle, Lianchun Xiao, Eugene J Koay

Faculty, Staff and Student Publications

BACKGROUND: Recent randomized controlled trials have established chemoimmunotherapy as the standard of care for patients with advanced biliary cancers with a median overall survival (OS) of about thirteen months. No data exist to demonstrate the safety and efficacy of liver-directed radiotherapy (RT) for extrahepatic and intrahepatic cholangiocarcinoma (CCA) in the era of chemoimmunotherapy. The purpose of this study is to report our early experience treating patients with CCA using RT and chemoimmunotherapy.

METHODS: Twenty-eight patients with CCA who received chemoimmunotherapy sequentially and/or concurrently with RT were retrospectively analyzed. The median biologic equivalent dose (BED) of RT was 84.0 [interquartile range …

Resolving Slc6a1 Variable Expressivity With Deep Clinical Phenotyping And Drosophila Models, Kristy L Jay, Nikhita Gogate, Paige I Hall, Kimberly M Ezell, Jonathan C Andrews, Sharayu V Jangam, Hongling Pan, Kelvin Pham, Ryan German, Vanessa Gomez, Emily Jellinek-Russo, Eric A Storch, Brain Gene Registry Consortium, Undiagnosed Diseases Network, Shinya Yamamoto, Oguz Kanca, Hugo J Bellen, Herman A Dierick, Joy D Cogan, John A Phillips, Rizwan Hamid, Thomas Cassini, Lynette Rives, Sumit Pruthi, Hua-Chang Chen, Jennifer E Posey, Michael F Wangler

Faculty, Staff and Students Publications

Variants in SLC6A1 result in a rare neurodevelopmental disorder characterized by a variable clinical presentation of symptoms including developmental delay, epilepsy, motor dysfunction, and autism spectrum disorder. SLC6A1 haploinsufficiency has been confirmed as the predominant pathway of SLC6A1-related neurodevelopmental disorder (SLC6A1-NDD); however, the molecular mechanism underlying the variable clinical presentation remains unclear. Here, through work of the Undiagnosed Diseases Network, we identify an individual with an inherited p.A334S variant of uncertain significance. To resolve this variant and better understand the variable expressivity associated with SLC6A1, we assess the phenotypes of the proband in comparison with a cohort of 13 individuals …

Pediatric Non-Down Syndrome Acute Megakaryoblastic Leukemia Patients Have Dismal Outcomes Irrespective Of Allogeneic Hematopoietic Stem Cell Transplant: A Single-Center Experience, Gabriela Llaurador, Matthew Willis, Michele S Redell, M Monica Gramatges, Andrea N Marcogliese, Swati Naik, Robert Krance, Erin Doherty, Alexandra M Stevens

Faculty, Staff and Students Publications

Background: Pediatric non-Down Syndrome Acute Megakaryoblastic Leukemia (non-DS-AMKL) is a rare subtype of Acute Myeloid Leukemia (AML) arising from primitive megakaryocytes and is associated with poor outcomes. Given its high incidence of relapse, this subpopulation of children is frequently referred for allogeneic hematopoietic stem cell transplant (allo-HSCT) in first complete remission (CR1).

Objectives: The objective of this study was to describe the clinical outcomes of non-DS-AMKL pediatric patients in a large, single-institution cohort.

Methods: A retrospective review of the medical records of thirty-six patients diagnosed with non-DS-AMKL treated at Texas Children's Hospital from 2000 to 2022 was conducted.

Results: Twenty-nine …

Reply To Fernández Et Al. Comment On "Goudra Et Al. Anesthesia For Bronchoscopy-An Update, Basavana Goudra, Lalitha Sundararaman, Prarthna Chandar, Michael Green

Department of Medicine Faculty Papers

No abstract provided.

Impact Of Surgical Refusal On Overall Survival In Patients With Rectal Cancer, Vishal Abhimutt Mahesh, Anjali Yadav, Harsheen Kaur Manaise, Berkay Demirors, Paola Berrios Jiminez, Angel Aguayo-Merly, Jade C. Bowers, Bansi P. Savaliya, Reed Popp, Ramin Shekouhi, Guido Chiriboga, Syeda Hoorulain Ahmed, Fatima Mubarak, Esinam Ekpeh, Emmanuel Gabriel

School of Medicine Faculty Publications

BACKGROUND/AIM: While surgery is a major component of treatment for managing rectal cancer, some individuals opt against it, potentially affecting their chances of survival. This study investigated the clinical and demographic elements linked to the decision to refuse surgery and assessed the potential impact of this decision on overall survival (OS). PATIENTS AND METHODS: We conducted a retrospective cohort study using the U.S. National Cancer Database to analyze factors linked to surgery refusal in patients with rectal cancer. Clinical, demographic and treatment characteristics were compared using Pearson chi-square and Wilcoxon rank-sum tests. RESULTS: Among the 115,066 patients with rectal cancer …

Cmc Arthroplasty For Treatment Of The Painful And Arthritic Thumb: A Review Of Surgical Treatment Options With Preferred Technique, Jon E. Hammarstedt, Yehuda A. Masturov, Sebastian D. Arango, Jacob Zeitlin, Samuel H. Payne, Mark S. Rekant

Department of Medicine Faculty Papers

Arthritis of the first carpometacarpal joint is a prevalent and often disabling condition for many older adults. The diagnosis is typically established through a comprehensive patient history, physical examination, and confirmatory radiographs. Nonsurgical management with splinting and anti-inflammatory medication has demonstrated effectiveness. Several surgical options have proven effectiveness for those who fail nonsurgical management. Surgical options include silicone implantation, resection arthroplasty, tendon interposition, ligament reconstruction, denervation, and joint implant arthroplasty. In the following review, we will elaborate upon the array of therapeutic approaches, culminating in the delineation of our preferred method.

Longitudinal Characterization Of Males With X-Linked Creatine Transporter Deficiency: Final Results Of A Multiyear Observational Study, Judith S Miller, Cristan Farmer, Susan Blair, Simona Bianconi, Natacha Akshoomoff, Irina Anselm, Bruce A Barshop, Lindsey Becker, Amanda E Bennett, Leandra N Berry, Elizabeth M Berry-Kravis, Aleksandra Bruchey, Anna W Byars, Tricia Cimms, Kim M Cecil, Maxine Covello, Laura S Cubit, Tanvi Das, Robert J Davis, Madison Drye, Can Ficicioglu, John B Fulton, Robin P Goin-Kochel, Whitney Guthrie, Barbara E Hallinan, Fady Hannah-Shmouni, Kathryn E Gustafson, Dwight D Koeberl, Nicola Longo, Eva Mamak, Saadet Mercimek-Andrews, Claire Michalak, Forbes D Porter, Samar Rahhal, Linda Rees, Gail A Spiridigliozzi, Caitlin Stone, Nancy R Sullivan, V Reid Sutton, Rebecca P Thomas, Manisha Udhnani, Susan Waisbren, Michelle Xu, Lin Zhang, Melanie Brandabur, Audrey Thurm

Faculty, Staff and Students Publications

Background: The purpose of the Vigilan observational study (ClinicalTrials.gov, NCT02931682) was to prospectively assess the natural history and developmental course of creatine transporter deficiency (CTD).

Methods: Males with CTD aged 6 months to 65 years were evaluated at 6-month intervals for up to 4 years. Evaluations included neurodevelopmental assessments of intellectual functioning, adaptive functioning, and challenging behaviors and the onset and progression of medical comorbidities.

Results: Fifty participants (median age, 7.6 years) were enrolled. The predominant CTD phenotype consisted of significant intellectual disabilities and limited skill development over time. Most participants had a history of febrile or nonfebrile seizures, …