Hematology Commons^™

Quiet A Puzzle: A Case Report Of 15 Year Old Female With Severe Anemia Due To Scurvy, Shaikha Alqahtani, Wang Zhihong

Graduate Medical Education (GME) Resident and Fellow Research Day Posters

Scurvy is historically thought of a disease affecting sailors in the seventeenth century. Vitamin C deficiency is rare in the developed world. It is mainly found in patients with poor nutrition and specifically those with developmental delay. The spectrum of scurvy is varied and includes dermatological, dental, bone and systemic manifestations. We present to you a case of a 15 year old female with a history of trisomy 21 presenting with severe anemia as a direct cause of vitamin C deficiency.

She presented with a one month history of progressive bilateral lower extremity bruising and inability to walk. Physical exam …

A Case Of Vancomycin-Induced Immune Thrombocytopenia., K. N. Macdougall, S. Parylo, A. Sokoloff

Journal Articles

Vancomycin-induced immune thrombocytopenia (ITP) is a rare, potentially life-threatening complication from an antibiotic frequently used in medical practice. We report a case of an 81-year-old male with recent removal of an infected right knee prosthesis and insertion of an articulating antibiotic spacer, presenting from rehabilitation for severe thrombocytopenia (1 X 103/µL). The patient's thrombocytopenia was initially falsely attributed to rifampin-induced ITP, a much more common cause of drug-induced thrombocytopenia. Only later, after a second precipitous drop in platelet count, vancomycin was correctly identified as the culprit. The patient's serum was tested for drug-dependent platelet antibodies with and without vancomycin. A …

Mutations Associated With A 17-Gene Leukemia Stem Cell Score And The Score’S Prognostic Relevance In The Context Of The European Leukemianet Classification Of Acute Myeloid Leukemia, M. Bill, D. Nicolet, J. Kohlschmidt, C. J. Walker, K. Mrózek, A. K. Eisfeld, D. Papaioannou, X. Rong-Mullins, J. E. Kolitz, C. D. Bloomfield, +8 Additional Authors

Journal Articles

© 2020 Ferrata Storti Foundation. Leukemia stem cells (LSC) are more resistant to standard chemotherapy and their persistence during remission can cause relapse, which is still one of the major clinical challenges in the treatment of acute myeloid leukemia (AML). A better understanding of the mutational patterns and the prognostic impact of molecular markers associated with stemness could lead to better clinical management and improve patients’ outcomes. We applied a previously described 17-gene expression score comprising genes differently expressed between LSC and leukemic bulk blasts, for 934 adult patients with de novo AML, and studied associations of the 17-gene LSC …

Advances In Targeted Therapy For Acute Myeloid Leukemia., Jifeng Yu, Peter Y Z Jiang, Hao Sun, Xia Zhang, Zhongxing Jiang, Yingmei Li, Yongping Song

Articles, Abstracts, and Reports

Acute myeloid leukemia (AML) is a clonal malignancy characterized by genetic heterogeneity due to recurrent gene mutations. Treatment with cytotoxic chemotherapy has been the standard of care for more than half of a century. Although much progress has been made toward improving treatment related mortality rate in the past few decades, long term overall survival has stagnated. Exciting developments of gene mutation-targeted therapeutic agents are now changing the landscape in AML treatment. New agents offer more clinical options for patients and also confer a more promising outcome. Since Midostaurin, a FLT3 inhibitor, was first approved by US FDA in 2017 …

The Impact Of Age On Survival In Cll Patients Receiving Ibrutinib As Initial Therapy., Chaitra Ujjani, Anthony Mato, Brian T Hill, John N Allan, Frederick Lansigan, Ryan Jacobs, Alan Skarbnik, Hande Tuncer, John M Pagel, Danielle Brander, Bruce Cheson, Paul Barr, Lindsey E Roeker, Jeffrey Pu, Nirav N Shah, Andre Goy, Stephen J Schuster, Nicole Lamanna, Alison Sehgal, Constantine S Tam, Mazyar Shadman

Articles, Abstracts, and Reports

Introduction: Recent randomized trials have demonstrated the efficacy of ibrutinib-based therapy in the treatment of patients with CLL. In Alliance A041202, a higher than expected number of unexplained deaths were reported with front-line ibrutinib in a patient population aged at least 65 years compared to ECOG 1912, which included patients up to 70 years of age.

Methods: Therefore, we conducted a retrospective analysis to investigate whether ibrutinib was associated with a greater mortality in older patients outside of a clinical trial setting. This multicenter analysis was performed by investigators at 20 academic and community practices.

Results: Amongst the 391 patients …

A Report From The Grndad Multi-Site Registry For Sickle Cell Disease: Iron Overload Is Under-Recognized And Under-Managed, Matthew Sears, Phd, Sophie Lanzkron, Md, Mhs, Carolyn Hoope, Md, Joshua J. Field, Md, E. Leila Jerome Clay, Md, Mcts, Faap, Susan Padrino, Md, Payal C. Desai, Md, Lynne D. Neumayr, Deepa Manwani, Md, Jane Little, Md

Phase 1

Introduction: GRNDaD is a prospective registry for people with SCD that opened to enrollment in 2016. Nine comprehensive SCD centers from across the United States are currently enrolling patients. The registry includes iron status and management data, important in SCD because chronic transfusion therapy is a mainstay of prophylactic management. Each unit of transfused blood introduces approximately 250 mg of iron into the blood, which can lead to systemic iron deposition, and untreated may lead to organ dysfunction or death.

Methods: GRNDaD currently contains prospective baseline and annual update information on approximately 1000 people with SCD. We analyzed ferritin levels …

Generation Of Induced Pluripotent Stem Cells From Blood Cell Of Glucose-6-Phosphate Dehydrogenase (G6pd) Deficiency, Malinee Tongbaen

Chulalongkorn University Theses and Dissertations (Chula ETD)

Glucose-6-phosphate dehydrogenase (G6PD) deficiency is the most common genetic enzyme disorder that affects more than 400 million people worldwide. Insufficient of G6PD enzyme or defect in enzyme activity can cause acute hemolysis after exposure to oxidative stress. The disease model used for pathogenesis study underlying oxidative damage or drug screening in G6PD deficient patients is still needed. In vitro generation of human-induced pluripotent stem cells (iPSCs) afford the potential source of the disease model in medical research. This study aimed to generate the iPSCs derived from G6PD deficient-subjects for used as a specific-disease model. The G6PD-derived iPSCs were generated from …

The Efficacy Of Acute Normovolemic Hemodilution Compared To Cell Salvage As A Blood Transfusion Alternative, Julia Yu

Capstone Showcase

Blood transfusions are currently the treatment of choice for significant blood loss. However, there are a variety of reasons for which they cannot be utilized. Current alternatives are acute normovolemic hemodilution (ANH) and cell 1 This study analyzes whether ANH or cell salvage is more effective in minimizing blood loss in perioperative or trauma patients. Multiple different studies involving either ANH or cell salvage were compared to each other. However, due to the lack of research conducted in either method, results obtained were largely inconclusive.

The Evolving Pharmacotherapeutic Landscape For The Treatment Of Sickle Cell Disease., Samir K Ballas

Cardeza Foundation for Hematologic Research

Sickle cell disease (SCD) is an extremely heterogeneous disease that has been associated with global morbidity and early mortality. More effective and inexpensive therapies are needed. During the last five years, the landscape of the pharmacotherapy of SCD has changed dramatically. Currently, 54 drugs have been used or under consideration to use for the treatment of SCD. These fall into 3 categories: the first category includes the four drugs (Hydroxyurea, L-Glutamine, Crizanlizumab tmca and Voxelotor) that have been approved by the United States Food and Drug Administration (FDA) based on successful clinical trials. The second category includes 22 drugs that …

Role Of Nurd In Acute Leukemia Cell Survival, Javeria Aijaz

Theses and Dissertations

Depletion of the ATPase component of the Nucleosome Remodeling and Deacetylase (NuRD) complex, CHD4, reduces acute myeloid leukemia (AML) cell survival. This study identified other NuRD components, as potential therapeutic targets for disrupting protein-protein interactions within NuRD. In addition to AML, we established that T-cell Acute Lymphoblastic Leukemia (T-ALL) cell lines responded similarly to CHD4 depletion.

Greater than 90% depletion of either MBD2 or MBD3 (the mutually exclusive two DNA binding NuRD paralogues) – was unremarkable, but complete depletion of MBD3 increased apoptosis and genotoxic sensitivity. Combined depletion of MBD-NuRD proteins augmented apoptosis observed with complete MBD3 depletion - indicating …

Prevalence And Predictors Of Chronic Pain Intensity And Disability Among Adults With Sickle Cell Disease, Nadine Matthie, Coretta Jenerette, Ashley Gibson, Sudeshna Paul, Melinda Higgins, Lakshmanan Krishnamurti

Faculty, Staff and Student Publications

Among 170 adults with sickle cell disease, we evaluated chronic pain impact and disability prevalence, assessed age and gender differences, and identified psychosocial predictors of chronic pain intensity and disability. Most participants had a high level of disability. Chronic pain intensity and disability were significantly associated with pain catastrophizing and chronic pain self-efficacy, and worsened with age. Further research is needed to confirm study findings and develop interventions, including palliative care approaches that address catastrophizing and disability, particularly for young women and middle-aged adults with sickle cell disease. Moreover, consistent clinical assessment of chronic pain and psychosocial health should be …

Educational Case: Systemic Mastocytosis With An Associated Hematological Neoplasm, Brenda Mai, Md A Wahed, Lei Chen, Nghia D Nguyen, Xiaohong Iris Wang, Zhihong Hu

Faculty, Staff and Student Publications

The following fictional case is intended as a learning tool within the Pathology Competencies for Medical Education (PCME), a set of national standards for teaching pathology. These are divided into three basic competencies: Disease Mechanisms and Processes, Organ System Pathology, and Diagnostic Medicine and Therapeutic Pathology. For additional information, and a full list of learning objectives for all three competencies, see http://journals.sagepub.com/doi/10.1177/2374289517715040.1

The Longitudinal Parallel Process Analysis Of Biomarkers Of Oxidative Stress, Symptom Clusters, And Cognitive Function In Children With Leukemia, Mary C Hooke, Daniel Hatch, Marilyn J Hockenberry, Susan Whitman, Ida Moore, David Montgomery, Kari Marano, Pauline Mitby, Michael E Scheurer, Olga Taylor, Wei Pan

Faculty, Staff and Students Publications

Background: During treatment for acute lymphoblastic leukemia (ALL), children report co-occurring symptoms of fatigue, sleep disturbance, pain, nausea, and depression as a symptom cluster. Central nervous system–directed ALL therapies also put children at risk for cognitive impairments. Cancer therapies can cause an increase in oxidative stress, which may contribute to treatment-related symptoms. This study examined the longitudinal relationships between biomarkers of oxidative stress in the cerebrospinal fluid, the Childhood Cancer Symptom Cluster–Leukemia (CCSC-L), and cognition, in children over the first year of ALL treatment. Methods: Glutathione (GSH) biomarkers of oxidative stress were measured in cerebrospinal fluid collected during treatment lumbar …

Acute Lymphoblastic Leukemia Presenting Initially As Spinal Cord Compression: When Chemotherapy Alone Is Enough, Albert Jang, Kallie N Kram, Scott N Berger, Mahmoud R Gaballa, Lee B Lu, David R Dunaway, Courtney N Miller-Chism

Faculty, Staff and Students Publications

Spinal cord compression (SCC) is a rare initial presentation and complication of acute lymphoblastic leukemia (ALL) with nearly all reported cases occurring in the pediatric population. We report a 38-year-old previously healthy man who presented with acute on chronic lower back pain, gait instability, urinary retention, and severe thrombocytopenia. Radiologic examination revealed two soft tissue masses of the thoracic spine associated with compression fractures causing spinal canal narrowing and cord compression. Bone marrow biopsy confirmed the diagnosis of ALL. Immediate initiation of high-dose corticosteroids and systemic chemotherapy resolved the patient's symptoms without radiation therapy or surgical intervention. After two courses …