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Full-Text Articles in Hematology

Knowledge Of Parents About Thalassemia In Tabuk City: A Cross-Sectional Study, Sawsan Mohammed Alblewi, Amirah M. Alatawi, Ehab Mahmoud Hanafy, Faisal Mayudh Althobaiti, Abdulelah Hamad Al-Balawi, Faisal Mohammed Alamrani, Yazeed Mahmoud Ajwah, Omar Muslih Albalawi, Mohammed Majed Alquayr, Sultan Ibrahim Alhamdi Aug 2026

Knowledge Of Parents About Thalassemia In Tabuk City: A Cross-Sectional Study, Sawsan Mohammed Alblewi, Amirah M. Alatawi, Ehab Mahmoud Hanafy, Faisal Mayudh Althobaiti, Abdulelah Hamad Al-Balawi, Faisal Mohammed Alamrani, Yazeed Mahmoud Ajwah, Omar Muslih Albalawi, Mohammed Majed Alquayr, Sultan Ibrahim Alhamdi

Makara Journal of Health Research

Background: Thalassemia is a common inherited blood disorder and remains a significant public health concern in Saudi Arabia. Parental knowledge is essential for effective prevention, screening, and early intervention. This study aimed to assess the knowledge of thalassemia among parents in Tabuk City, Saudi Arabia, and identify factors associated with knowledge levels.

Methods: A cross-sectional study was conducted among 392 parents residing in Tabuk City between March and May 2025. Data were collected using a validated Arabic questionnaire distributed online. Knowledge was assessed using 21 items covering general thalassemia knowledge, thalassemia major, carrier status, and prevention. Knowledge levels …


Factors Associated With Pre-Donation Health-Related Quality-Of-Life Among Pediatric Sibling Hematopoietic Cell Donors: A Donorkids Ql Study., Galen E. Switzer, Jessica G. Bruce, Bronwen E. Shaw, Vidya Kuniyil, James W. Varni, Brandan J. Butler, Connor Erickson, Alisha Mussetter, Allison Neutzling, Hisham Abdel-Azim, Paibel Aguayo-Hiraldo, Eric J. Anderson, Victor M. Aquino, Karlie Boone, Farid Boulad, Joseph H. Chewning, Jessica Cooper, Ann Dahlberg, Christopher C. Dvorak, Jorge Galvez-Silva, Ann E. Haight, Jennifer A. Hoag, Michelle Hudspeth, David Jacobsohn, Kimberly A. Kasow, Carrie L. Kitko, Lakshmanan Krishnamurti, Lisa Madden, Holly K. Miller, Erin Morales, Timothy S. Olson, Anna B. Pawlowska, Vinod K. Prasad, Troy C. Quigg, Andromachi Scaradavou, Niketa C. Shah, Shalini Shenoy, Nancy Terwilliger, Lori Wiener, Gregory A. Yanik, Lolie C. Yu, Michael A. Pulsipher Jul 2026

Factors Associated With Pre-Donation Health-Related Quality-Of-Life Among Pediatric Sibling Hematopoietic Cell Donors: A Donorkids Ql Study., Galen E. Switzer, Jessica G. Bruce, Bronwen E. Shaw, Vidya Kuniyil, James W. Varni, Brandan J. Butler, Connor Erickson, Alisha Mussetter, Allison Neutzling, Hisham Abdel-Azim, Paibel Aguayo-Hiraldo, Eric J. Anderson, Victor M. Aquino, Karlie Boone, Farid Boulad, Joseph H. Chewning, Jessica Cooper, Ann Dahlberg, Christopher C. Dvorak, Jorge Galvez-Silva, Ann E. Haight, Jennifer A. Hoag, Michelle Hudspeth, David Jacobsohn, Kimberly A. Kasow, Carrie L. Kitko, Lakshmanan Krishnamurti, Lisa Madden, Holly K. Miller, Erin Morales, Timothy S. Olson, Anna B. Pawlowska, Vinod K. Prasad, Troy C. Quigg, Andromachi Scaradavou, Niketa C. Shah, Shalini Shenoy, Nancy Terwilliger, Lori Wiener, Gregory A. Yanik, Lolie C. Yu, Michael A. Pulsipher

Manuscripts, Articles, Book Chapters and Other Papers

Due to limited published data assessing pediatric hematopoietic cell donor experiences, we previously conducted one of the largest quantitative investigations of pediatric donor experiences and health-related quality-of-life (HRQoL) at the time (RDSafe). Findings from RDSafe demonstrated that a subset of pediatric HC donors experienced very poor HRQoL; unfortunately, that dataset addressed only a limited number of factors, and key associations explaining this poor HRQoL were not found. In this study, our goal was to address that deficit by describing pre-donation donor HRQoL in detail and identifying factors across five key domains that were associated with donor HRQoL. We conducted a …


Hydroxyurea Pharmacokinetics In Children With Sickle Cell Anemia Across Different Global Populations, Alexandra Power-Hays, Kathryn Mcelhinney, Thomas Williams, George Mochamah, Peter Olupot-Olupot, George Paasi, Marvin Reid, Angela Rankine-Mullings, Robert Opoka, John Chandy Jan 2026

Hydroxyurea Pharmacokinetics In Children With Sickle Cell Anemia Across Different Global Populations, Alexandra Power-Hays, Kathryn Mcelhinney, Thomas Williams, George Mochamah, Peter Olupot-Olupot, George Paasi, Marvin Reid, Angela Rankine-Mullings, Robert Opoka, John Chandy

Paediatrics and Child Health, East Africa

Hydroxyurea provides effective disease-modifying treatment for people with sickle cell anemia (SCA), especially when escalated to maximum tolerated dose (MTD), but has wide interpatient dosing variability due to pharmacokinetic (PK) differences. Whether hydroxyurea PK parameters differ among children with SCA in different global regions is unknown. We compared hydroxyurea PK parameters among children with SCA from five clinical trials: HUSTLE (USA, NCT00305175), TREAT (USA, NCT02286154), NOHARM (Uganda, NCT01976416), REACH (Uganda and Kenya, NCT01966731), and EXTEND (Jamaica, NCT02556099). Key hydroxyurea PK parameters were determined using HdxSim™, a validated hydroxyurea PK-software program. The results were compared across regions by one way analysis …


Leukemia, Children's Health School Services Jan 2026

Leukemia, Children's Health School Services

School Guides

Educator guide about how to support students with leukemia.


Sickle Cell Disease, Children's Health School Services Jan 2026

Sickle Cell Disease, Children's Health School Services

School Guides

Educator guide about how to support students with sickle cell disease.


Growth Measurements In Ugandan Children With Sickle Cell Anaemia From A Hydroxyurea (Hydroxycarbamide) Treatment Trial Relative To Unaffected Sibling Controls, Dennis Kalibbala, Vincent Mboizi, Grace Nambatya, Susan Murungi, Joan Ashaba, Catherine Nabaggala, Lynnth Turyagyenda, Deogratias Munube, Phillip Kasirye, Robert Opoka Oct 2025

Growth Measurements In Ugandan Children With Sickle Cell Anaemia From A Hydroxyurea (Hydroxycarbamide) Treatment Trial Relative To Unaffected Sibling Controls, Dennis Kalibbala, Vincent Mboizi, Grace Nambatya, Susan Murungi, Joan Ashaba, Catherine Nabaggala, Lynnth Turyagyenda, Deogratias Munube, Phillip Kasirye, Robert Opoka

Paediatrics and Child Health, East Africa

No abstract provided.


Building Access To Care For Women And Girls+ With Bleeding Disorders: The Wgbd Clinic Of Excellence Model, Ming Y. Lim, Katherine C. Anguiano, Shannon L. Carpenter, Kerry B. Funkhouser Oct 2025

Building Access To Care For Women And Girls+ With Bleeding Disorders: The Wgbd Clinic Of Excellence Model, Ming Y. Lim, Katherine C. Anguiano, Shannon L. Carpenter, Kerry B. Funkhouser

Manuscripts, Articles, Book Chapters and Other Papers

No abstract provided.


Antithrombotic Strategies And Outcomes In Neonates And Infants With Cardiac Shunts: A Systematic Review And Meta-Analysis., Amy L. Kiskaddon, Neil A. Goldenberg, Marisol Betensky, Joshua W. Branstetter, Dina Ashour, Pamela Williams, Arabela C. Stock, Michael Silvey, Therese M. Giglia, Nhue L. Do, Cardiac Disease Thrombosis And Hemostasis Working Party Of The Isth Ssc Subcommittee On Pediatric And Neonatal Thrombosis And Hemostasis Aug 2025

Antithrombotic Strategies And Outcomes In Neonates And Infants With Cardiac Shunts: A Systematic Review And Meta-Analysis., Amy L. Kiskaddon, Neil A. Goldenberg, Marisol Betensky, Joshua W. Branstetter, Dina Ashour, Pamela Williams, Arabela C. Stock, Michael Silvey, Therese M. Giglia, Nhue L. Do, Cardiac Disease Thrombosis And Hemostasis Working Party Of The Isth Ssc Subcommittee On Pediatric And Neonatal Thrombosis And Hemostasis

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Cardiac shunt thrombosis in neonates and infants remains a concern for shunt failure and mortality. The optimal strategy for thromboprophylaxis remains unknown.

OBJECTIVES: This systematic review aims to characterize antithrombotic strategies and outcomes in neonates and infants with a cardiac shunt.

METHODS: MEDLINE, Embase, and Cochrane CENTRAL were searched from inception through July 2024 for studies reporting shunt thrombosis prevalence among infants who received a cardiac shunt. We estimated the pooled prevalence of shunt thrombosis using random-effects meta-analysis. In the subgroup analysis, we evaluated the effects of shunt type and antithrombotic strategies on shunt thrombosis prevalence.

RESULTS: A total …


American Society Of Hematology/International Society On Thrombosis And Haemostasis 2024 Updated Guidelines For Treatment Of Venous Thromboembolism In Pediatric Patients, Paul Monagle, Muayad Azzam, Rachel Bercovitz, Marisol Betensky, Rukhmi Bhat, Tina Biss, Brian Branchford, Leonardo R. Brandão, Anthony K.C. Chan, Vincent E.S. Faustino, Julie Jaffray, Sophie Jones, Hassan Kawtharany, Bryce A. Kerlin, Nicole Kucine, Riten Kumar, Christoph Male, Marie Claude Pelland-Marcotte, Leslie Raffini, Chittalsinh Raulji, Sarah E. Sartain, Clifford M. Takemoto, Cristina Tarango, C. Heleen Van Ommen, Maria C. Velez, Sara K. Vesely, John Wiernikowski, Suzan Williams, Hope P. Wilson, Et Al May 2025

American Society Of Hematology/International Society On Thrombosis And Haemostasis 2024 Updated Guidelines For Treatment Of Venous Thromboembolism In Pediatric Patients, Paul Monagle, Muayad Azzam, Rachel Bercovitz, Marisol Betensky, Rukhmi Bhat, Tina Biss, Brian Branchford, Leonardo R. Brandão, Anthony K.C. Chan, Vincent E.S. Faustino, Julie Jaffray, Sophie Jones, Hassan Kawtharany, Bryce A. Kerlin, Nicole Kucine, Riten Kumar, Christoph Male, Marie Claude Pelland-Marcotte, Leslie Raffini, Chittalsinh Raulji, Sarah E. Sartain, Clifford M. Takemoto, Cristina Tarango, C. Heleen Van Ommen, Maria C. Velez, Sara K. Vesely, John Wiernikowski, Suzan Williams, Hope P. Wilson, Et Al

School of Medicine Faculty Publications

Background: The American Society of Hematology (ASH) guidelines on treatment of pediatric venous thromboembolism (VTE) were published in 2018. In the last 6 years, there has been a 10-fold increase in the number of children involved in VTE treatment trials. Objective: The ASH Committee on Quality and Guidelines agreed to update the pediatric guidelines in conjunction with the International Society on Thrombosis and Haemostasis (ISTH). These ASH/ISTH evidence-based guidelines are intended to support patients, clinicians, and other health care professionals in the management of pediatric patients with VTE. Methods: ASH/ISTH formed a multidisciplinary guideline panel to minimize potential bias from …


Novel Rpl30 Variant In Diamond Blackfan Anemia Demonstrates Early Impact On Erythroid Differentiation With Downregulated Gata1-Hsp70, Alexandra Prosser-Dombrowski, Irina Pushel, Jacqelyn Nemechek, Priyanka Kumar, Jay L. Vivian, Jennifer Gerton, John M. Perry May 2025

Novel Rpl30 Variant In Diamond Blackfan Anemia Demonstrates Early Impact On Erythroid Differentiation With Downregulated Gata1-Hsp70, Alexandra Prosser-Dombrowski, Irina Pushel, Jacqelyn Nemechek, Priyanka Kumar, Jay L. Vivian, Jennifer Gerton, John M. Perry

Research Days

Background: Diamond Blackfan anemia (DBA) is an inherited bone marrow failure syndrome caused by ribosomal protein gene mutations leading to apoptosis of erythroid progenitors. We identified a novel heterozygous variant (c.167+769C>T) in the noncoding region of RPL30 in a patient diagnosed with DBA.

Objectives/Goal: We hypothesized that this variant stunts erythroid differentiation at the proerythroblast stage and is pathogenic for DBA.

Methods/Design: We developed an induced pluripotent stem cell (iPSC) model with a wild type (WT) and three CRISPR-Cas9 edited RPL30 mutant clones. iPSCs were differentiated into hematopoietic stem cells, which were assessed by flow cytometry and single cell …


Effect Of Social Determinants Of Health On Clinic Visit Attendance In Patients With Hemophilia, Shailly Gaur Md, Brian Lee Phd, Mph, James Anderst Md, Msci, Katie Foote Lscsw, Lcsw, Osw-C, Andrea Bradley-Ewing Mpa, Ma, Shannon Carpenter Md May 2025

Effect Of Social Determinants Of Health On Clinic Visit Attendance In Patients With Hemophilia, Shailly Gaur Md, Brian Lee Phd, Mph, James Anderst Md, Msci, Katie Foote Lscsw, Lcsw, Osw-C, Andrea Bradley-Ewing Mpa, Ma, Shannon Carpenter Md

Research Days

Background

Social determinants of health (SDOH) create barriers to seeking care regularly, especially for patients with chronic disease. Patients with moderate-severe hemophilia A and B (factor 8 and 9 deficiencies respectively) have a life-long higher risk of bleeding and require chronic therapies. Specialized care is offered through hemophilia treatment centers (HTCs); however, these can be difficult to access for some individuals. Previous SDOH research in this patient population has been limited; therefore, it can be difficult to identify the barriers to care that exist. There is a need to examine SDOH more thoroughly to create patient-directed interventions to improve access …


Respiratory Failure In Pediatric Hematology And Oncology Patients On Extracorporeal Membrane Oxygenation: A Comparative Analysis, Michelle Brown Do, Jenna Miller, Asdis Finnsdottir Wagner, Erin Hall, Maya Dewan Md, Christopher Dandoy Md, Kalee Grassia Md, Bin Zhang Phd May 2025

Respiratory Failure In Pediatric Hematology And Oncology Patients On Extracorporeal Membrane Oxygenation: A Comparative Analysis, Michelle Brown Do, Jenna Miller, Asdis Finnsdottir Wagner, Erin Hall, Maya Dewan Md, Christopher Dandoy Md, Kalee Grassia Md, Bin Zhang Phd

Research Days

This project was a retrospective comparative analysis between two tertiary care pediatric institutions. The primary objective entailed examining survival outcomes in the oncologic and hematopoetic stem cell transplant patient populations with respiratory failure in the presence and absence of ECMO support.


Increasing Incentive Spirometry Use In Patients With Sickle Cell Disease Admitted To Cmh, Juan Nario Ramirez, Alexandra Prosser-Dombrowski, Thomas R. Cochran, Joel Thompson May 2025

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 …


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 Mar 2025

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.


Clinical Relapse Versus Treatment Failure: The Case For Surveillance For Re-Appearance Of Minimal Measurable Disease In Pediatric Patients With Higher Risk B-All, Paul S. Gaynon, Linwei Li Mar 2025

Clinical Relapse Versus Treatment Failure: The Case For Surveillance For Re-Appearance Of Minimal Measurable Disease In Pediatric Patients With Higher Risk B-All, Paul S. Gaynon, Linwei Li

Research Symposium

Background: Despite significant advancements in the treatment of pediatric B-cell acute lymphoblastic leukemia (B-ALL), chemotherapy has reached its end of “intensification” stage despite improvements in supportive care. Moreover, relapse remains a major challenge, particularly in high-risk populations such as adolescents and young adults (AYAs). The definition of threshold for clinical relapse as 25% presence of marrow lymphoblasts was established decades ago, which may be incoherent with current therapeutic strategies and delay the window for timely treatment for relapsed patients. Emerging data suggest that early detection of minimal residual disease (MRD) may offer an opportunity to intervene before clinical relapse, improving …


Neurocognitive Gains Among Ugandan Children With Sickle Cell Anemia On Hydroxyurea: 18-Month Trial Interim Results, Shubaya Kasule Naggayi, Dennis Kalibbala, Vincent Mboizi, John M. Ssenkusu, Zhezhen Jin, Caterina Rosano, Deogratias Munube, Bill Wambaka, Ruth Namazzi, Robert Opoka Mar 2025

Neurocognitive Gains Among Ugandan Children With Sickle Cell Anemia On Hydroxyurea: 18-Month Trial Interim Results, Shubaya Kasule Naggayi, Dennis Kalibbala, Vincent Mboizi, John M. Ssenkusu, Zhezhen Jin, Caterina Rosano, Deogratias Munube, Bill Wambaka, Ruth Namazzi, Robert Opoka

Paediatrics and Child Health, East Africa

Children with sickle cell anemia (SCA) frequently develop progressive neurocognitive impairment. We aimed to determine effects of hydroxyurea therapy on neurocognitive function in Ugandan children with SCA by comparing levels at enrollment to a planned 18-month interim assessment. Ugandan children (N=264) ages 3-9 years were enrolled from a SCA clinic and treated in a 30-month singlearm open-label trial with escalation to maximum tolerated dose (MTD). Primary outcome was the effects of hydroxyurea on cognition, attention and executive function, along with transcranial doppler ultrasound (TCD) blood flow velocity. Sibling controls (N=110) without SCA underwent neurocognitive testing in parallel to establish age-normalized …


Effect Of Social Determinants Of Health On Clinic Visit Attendance In Patients With Hemophilia, Shailly Gaur, Brian Lee Phd, Mph, James Anderst Md, Msci, Katie Foote Lscsw, Lcsw, Osw-C, Andrea Bradley-Ewing Mpa, Ma, Shannon L. Carpenter Mar 2025

Effect Of Social Determinants Of Health On Clinic Visit Attendance In Patients With Hemophilia, Shailly Gaur, Brian Lee Phd, Mph, James Anderst Md, Msci, Katie Foote Lscsw, Lcsw, Osw-C, Andrea Bradley-Ewing Mpa, Ma, Shannon L. Carpenter

Posters

Background Social determinants of health (SDOH) create barriers to seeking care regularly, especially for patients with chronic disease. Patients with moderate-severe hemophilia A and B (factor 8 and 9 deficiencies respectively) have a life-long higher risk of bleeding and require chronic therapies. Specialized care is offered through hemophilia treatment centers (HTCs); however, these can be difficult to access for some individuals. Previous SDOH research in this patient population has been limited; therefore, it can be difficult to identify the barriers to care that exist. There is a need to examine SDOH more thoroughly to create patient-directed interventions to improve access …


The Feasibility Of Pharmacokinetic-Based Dosing Of Hydroxyurea For Children With Sickle Cell Anaemia In Uganda : Baseline Results Of The Alternative Dosing And Prevention Of Transfusions Trial, Alexandra Power Hays, Ruth Namazzi, Min Dong, Caroline Kazinga, Charles Kato, Sadat Aliwuya, Kathryn Mcelhinney, Andrea L. Conroy, Adam Lane, Robert Opoka Mar 2025

The Feasibility Of Pharmacokinetic-Based Dosing Of Hydroxyurea For Children With Sickle Cell Anaemia In Uganda : Baseline Results Of The Alternative Dosing And Prevention Of Transfusions Trial, Alexandra Power Hays, Ruth Namazzi, Min Dong, Caroline Kazinga, Charles Kato, Sadat Aliwuya, Kathryn Mcelhinney, Andrea L. Conroy, Adam Lane, Robert Opoka

Paediatrics and Child Health, East Africa

Pharmacokinetic (PK)-guided dosing of hydroxyurea for children with sickle cell anaemia (SCA) could optimize dosing and improve outcomes, but its feasibility has not been demonstrated in low-resource settings where the majority of affected children live. Alternative Dosing And Prevention of Transfusions (ADAPT) is a prospective trial evaluating blood transfusions and the feasibility of determining PK-guided, hydroxyurea maximum tolerated doses (MTD) for children with SCA in Uganda, using portable high-performance liquid chromatography (HPLC) and a novel PK software programme (HdxSim). ADAPT enrolled 106 participants, and 100% completed PK testing. PK-guided doses were generated for 78%, of which 38% were within the …


Hyperleukocytosis In A Neuroblastoma Patient After Treatment With Natural Killer T Cells Expressing A Gd2-Specific Chimeric Antigen Receptor And Il-15, Gengwen Tian, Amy N Courtney, Hangjin Yu, Saleh Bhar, Xin Xu, Gabriel A Barragán, Claudia Martinez Amador, Nisha Ghatwai, Michael S Wood, Deborah Schady, Antonino Montalbano, Shantan Reddy, Aoife M Roche, David De La Cerda, Donald Williams Parsons, Erica J Di Pierro, Frederic D Bushman, Andras Heczey, Leonid S Metelitsa Jan 2025

Hyperleukocytosis In A Neuroblastoma Patient After Treatment With Natural Killer T Cells Expressing A Gd2-Specific Chimeric Antigen Receptor And Il-15, Gengwen Tian, Amy N Courtney, Hangjin Yu, Saleh Bhar, Xin Xu, Gabriel A Barragán, Claudia Martinez Amador, Nisha Ghatwai, Michael S Wood, Deborah Schady, Antonino Montalbano, Shantan Reddy, Aoife M Roche, David De La Cerda, Donald Williams Parsons, Erica J Di Pierro, Frederic D Bushman, Andras Heczey, Leonid S Metelitsa

Faculty, Staff and Students Publications

The ability of immune cells to expand numerically after infusion distinguishes adoptive immunotherapies from traditional drugs, providing unique therapeutic advantages as well as the potential for unmanageable toxicities. Here, we describe a case of lethal hyperleukocytosis in a patient with neuroblastoma treated on phase 1 clinical trial (NCT03294954) with autologous natural killer T cells (NKTs) expressing a GD2-specific chimeric antigen receptor and cytokine interleukin 15 (GD2-CAR.15). This patient was the first to be treated on dose level (DL) 5 and the first patient whose product was restimulated with K562-derived artificial antigen-presenting cells (aAPCs) instead of autologous peripheral blood mononuclear cells …


Association Of Unmet Social Needs With Disease-Related Outcomes In Pediatric Patients With Sickle Cell Disease, Molly Sonenklar, Sarah Marks, Cerelia Donald, Cecelia Valrie, Wally Smith, India Sisler Dec 2024

Association Of Unmet Social Needs With Disease-Related Outcomes In Pediatric Patients With Sickle Cell Disease, Molly Sonenklar, Sarah Marks, Cerelia Donald, Cecelia Valrie, Wally Smith, India Sisler

School of Medicine Faculty Publications

Background: Social determinants of health (SDoH) are socioeconomic factors that influence health and well-being, though when unmet can greatly contribute to health disparities. Individuals with sickle cell disease (SCD) are at increased risk of mortality, disability, and healthcare utilization. However, there are limited data linking specific social needs with disease outcomes in this population. Therefore, we sought to identify the unmet needs in one institution and their association with healthcare utilization. Methods: Children with SCD and their guardians answered demographic and SDoH questionnaires during routine Sickle Cell Clinic appointments. We defined an unmet social need as any positive answer to …


Eculizumab For Management Of Hyperhemolysis Syndrome In Pediatric Patients With Sickle Cell Disease: A Single-Center Case Series, Ajibike Lapite, Saleh Bhar, Titilope Fasipe Aug 2024

Eculizumab For Management Of Hyperhemolysis Syndrome In Pediatric Patients With Sickle Cell Disease: A Single-Center Case Series, Ajibike Lapite, Saleh Bhar, Titilope Fasipe

Faculty, Staff and Students Publications

Chronic hemolytic anemia and vascular occlusion are hallmarks of sickle cell disease (SCD). Blood transfusions are critical for supportive and preventive management of SCD complications. Patients with SCD are at risk for hyperhemolysis syndrome (HHS), a subtype of delayed hemolytic transfusion reactions. HHS management includes intravenous immunoglobulin, corticosteroids, and avoidance of further transfusions. Not all patients respond to first-line agents. Eculizumab, which blocks terminal complement activation, has been proposed as second-line management of HHS. We describe two patients who received eculizumab for refractory HHS. In our experience, eculizumab is a safe and effective option for refractory pediatric HHS.


An Investigation Into Aetiology, Detection And Treatment Of Neonatal Alloimmune Thrombocytopenia, Mairead Horan Jul 2024

An Investigation Into Aetiology, Detection And Treatment Of Neonatal Alloimmune Thrombocytopenia, Mairead Horan

International Undergraduate Journal of Health Sciences

NAIT is a rare disorder with a similar aetiology to HDN, however unlike its erythrocyte counterpart, thrombocyte immunisation can occur within the first pregnancy. (Giouleka et al., 2023). The most common antibodies implicated are HLA-1a. (Winkelhorst et al., 2017). 2.5% of the Caucasian population are HPA-1a negative, of this population 33% are HLA-DR-B3*0101 positive increasing the risk of producing an alloantibody upon encountering the HPA-1a antigen. The maternal system becomes alloimmunised to the foreign paternal antigens of the foetus/neonate, which cross the placenta causing low platelets of the foetus. (Giouleka et al., 2023).

A third of antigen-positive neonates born to …


Early Cholestasis And Vitamin K Deficiency Secondary To Biliary Atresia Presenting As Coagulopathy And Thymic Hemorrhage, Abbey Elsbernd, Lauren Amos Md May 2024

Early Cholestasis And Vitamin K Deficiency Secondary To Biliary Atresia Presenting As Coagulopathy And Thymic Hemorrhage, Abbey Elsbernd, Lauren Amos Md

Research Days

Background

Bleeding and progressive coagulopathy are established symptoms of early cholestasis, including extrahepatic biliary atresia. We present a patient with bleeding secondary to vitamin K deficiency and cholestasis-induced fat malabsorption with unusual presentation of mediastinal mass.

Objective

Describe a case of late-onset vitamin K deficiency and cholestasis presenting as jaundice and coagulopathy with associated bleeding into thymic structures Design/Method

Case report

Results

A 4-week-old term male infant was brought to the ED with fussiness and bruising. Prior to presentation, he was afebrile and followed regularly with his primary care provider for jaundice below phototherapy thresholds. Infant received vitamin K at …


Testing For Bleeding Disorders In Child Abuse: Adherence To Aap Recommendations And Results Of Testing., Lyndsey Hultman, James Anderst, Henry T. Puls, Shannon L. Carpenter, Angela Doswell, Angela N. Bachim, Joanne Wood, Kristine Campbell, Daniel M. Lindberg May 2024

Testing For Bleeding Disorders In Child Abuse: Adherence To Aap Recommendations And Results Of Testing., Lyndsey Hultman, James Anderst, Henry T. Puls, Shannon L. Carpenter, Angela Doswell, Angela N. Bachim, Joanne Wood, Kristine Campbell, Daniel M. Lindberg

Research Days

This was a retrospective, descriptive study of bleeding disorder testing practices among subjects in CAPNET, a multicenter research network of children evaluated by Child Abuse Pediatricians for concern of child physical abuse.


Retrospective Analysis Of Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome In Paediatric Patients Undergoing Hematopoietic Cell Transplantation -A Multicentre Study, Dristhi Ragoonanan, Hisham Abdel-Azim, Aditya Sharma, Saleh Bhar, Jennifer Mcarthur, Renee Madden, April Rahrig, Rajinder Bajwa, Jian Wang, Victoria Sun, Mariah Wright, Rebekah Lassiter, Basirat Shoberu, Jitesh Kawedia, Sajad Jawad Khazal, Kris Michael Mahadeo, Pediatric Acute Lung Injury And Sepsis Investigators (Palisi) Network May 2024

Retrospective Analysis Of Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome In Paediatric Patients Undergoing Hematopoietic Cell Transplantation -A Multicentre Study, Dristhi Ragoonanan, Hisham Abdel-Azim, Aditya Sharma, Saleh Bhar, Jennifer Mcarthur, Renee Madden, April Rahrig, Rajinder Bajwa, Jian Wang, Victoria Sun, Mariah Wright, Rebekah Lassiter, Basirat Shoberu, Jitesh Kawedia, Sajad Jawad Khazal, Kris Michael Mahadeo, Pediatric Acute Lung Injury And Sepsis Investigators (Palisi) Network

Faculty, Staff and Students Publications

BACKGROUND: Sinusoidal obstruction syndrome is a potentially fatal complication following hematopoietic cell transplantation, high-intensity chemotherapies and increasingly seen with calicheamicin based leukemia therapies. Paediatric specific European Society for Blood and Marrow Transplantation (pEBMT) diagnostic criteria have demonstrated benefit in single center studies compared to historic criteria. Yet, the extent to which they have been universally implemented remains unclear.

METHODS: We conducted a retrospective multi-centre study to examine the potential impact of the Baltimore, modified Seattle and pEBMT criteria on the incidence, severity, and outcomes of sinusoidal obstruction syndrome among paediatric hematopoietic cell transplantation patients.

FINDINGS: The incidence of sinusoidal obstruction …


The Evidence Is Mounting: Insurance Coverage Disruptions Lead To Worse Outcomes Among Children Diagnosed With Cancer, Philip J Lupo, Christina L Magyar May 2024

The Evidence Is Mounting: Insurance Coverage Disruptions Lead To Worse Outcomes Among Children Diagnosed With Cancer, Philip J Lupo, Christina L Magyar

Faculty, Staff and Students Publications

No abstract provided.


Antiviral Cellular Therapy For Enhancing T-Cell Reconstitution Before Or After Hematopoietic Stem Cell Transplantation (Aces): A Two-Arm, Open Label Phase Ii Interventional Trial Of Pediatric Patients With Risk Factor Assessment., Michael D. Keller, Patrick J. Hanley, Yueh-Yun Chi, Paibel Aguayo-Hiraldo, Christopher C. Dvorak, Michael R. Verneris, Donald B. Kohn, Sung-Yun Pai, Blachy J. Dávila Saldaña, Benjamin Hanisch, Troy C. Quigg, Roberta H. Adams, Ann Dahlberg, Shanmuganathan Chandrakasan, Hasibul Hasan, Jemily Malvar, Mariah A. Jensen-Wachspress, Christopher A. Lazarski, Gelina Sani, John M. Idso, Haili Lang, Pamela Chansky, Chase D. Mccann, Jay Tanna, Allistair A. Abraham, Jennifer L. Webb, Abeer Shibli, Amy K. Keating, Prakash Satwani, Pawel Muranski, Erin Hall, Michael J. Eckrich, Evan Shereck, Holly Miller, Ewelina Mamcarz, Rajni Agarwal, Satiro N. De Oliveira, Mark T. Vander Lugt, Christen L. Ebens, Victor M. Aquino, Jeffrey J. Bednarski, Julia Chu, Suhag Parikh, Jennifer Whangbo, Michail Lionakis, Elias T. Zambidis, Elizabeth Gourdine, Catherine M. Bollard, Michael A. Pulsipher Apr 2024

Antiviral Cellular Therapy For Enhancing T-Cell Reconstitution Before Or After Hematopoietic Stem Cell Transplantation (Aces): A Two-Arm, Open Label Phase Ii Interventional Trial Of Pediatric Patients With Risk Factor Assessment., Michael D. Keller, Patrick J. Hanley, Yueh-Yun Chi, Paibel Aguayo-Hiraldo, Christopher C. Dvorak, Michael R. Verneris, Donald B. Kohn, Sung-Yun Pai, Blachy J. Dávila Saldaña, Benjamin Hanisch, Troy C. Quigg, Roberta H. Adams, Ann Dahlberg, Shanmuganathan Chandrakasan, Hasibul Hasan, Jemily Malvar, Mariah A. Jensen-Wachspress, Christopher A. Lazarski, Gelina Sani, John M. Idso, Haili Lang, Pamela Chansky, Chase D. Mccann, Jay Tanna, Allistair A. Abraham, Jennifer L. Webb, Abeer Shibli, Amy K. Keating, Prakash Satwani, Pawel Muranski, Erin Hall, Michael J. Eckrich, Evan Shereck, Holly Miller, Ewelina Mamcarz, Rajni Agarwal, Satiro N. De Oliveira, Mark T. Vander Lugt, Christen L. Ebens, Victor M. Aquino, Jeffrey J. Bednarski, Julia Chu, Suhag Parikh, Jennifer Whangbo, Michail Lionakis, Elias T. Zambidis, Elizabeth Gourdine, Catherine M. Bollard, Michael A. Pulsipher

Manuscripts, Articles, Book Chapters and Other Papers

Viral infections remain a major risk in immunocompromised pediatric patients, and virus-specific T cell (VST) therapy has been successful for treatment of refractory viral infections in prior studies. We performed a phase II multicenter study (NCT03475212) for the treatment of pediatric patients with inborn errors of immunity and/or post allogeneic hematopoietic stem cell transplant with refractory viral infections using partially-HLA matched VSTs targeting cytomegalovirus, Epstein-Barr virus, or adenovirus. Primary endpoints were feasibility, safety, and clinical responses (>1 log reduction in viremia at 28 days). Secondary endpoints were reconstitution of antiviral immunity and persistence of the infused VSTs. Suitable VST …


Investigating Markers For Intracranial Hemorrhage Severity: Insights From Cbc Changes And Beyond, Mckenzie Locker, Kasey Chelemedos, Nataly Apollonsky Apr 2024

Investigating Markers For Intracranial Hemorrhage Severity: Insights From Cbc Changes And Beyond, Mckenzie Locker, Kasey Chelemedos, Nataly Apollonsky

Tower Health Research Day

No abstract provided.


Early Cholestasis And Vitamin K Deficiency Secondary To Biliary Atresia Presenting As Coagulopathy And Thymic Hemorrhage, Abbey Elsbernd, Lauren Amos Md Apr 2024

Early Cholestasis And Vitamin K Deficiency Secondary To Biliary Atresia Presenting As Coagulopathy And Thymic Hemorrhage, Abbey Elsbernd, Lauren Amos Md

Posters

Background Bleeding and progressive coagulopathy are established symptoms of early cholestasis, including extrahepatic biliary atresia. We present a patient with bleeding secondary to vitamin K deficiency and cholestasis-induced fat malabsorption with unusual presentation of mediastinal mass. Objective Describe a case of late-onset vitamin K deficiency and cholestasis ¬¬presenting as jaundice and coagulopathy with associated bleeding into thymic structures Design/Method Case report Results A 4-week-old term male infant was brought to the ED with fussiness and bruising. Prior to presentation, he was afebrile and followed regularly with his primary care provider for jaundice below phototherapy thresholds. Infant received vitamin K at …


Single Center Retrospective Comparison Of Bivalirudin And Heparin For Therapeutic Anticoagulation In Pediatric Patients, Clayton Habiger, Shannon L. Carpenter Apr 2024

Single Center Retrospective Comparison Of Bivalirudin And Heparin For Therapeutic Anticoagulation In Pediatric Patients, Clayton Habiger, Shannon L. Carpenter

Posters

Background: The pharmacological choice for continuous anticoagulation therapy in pediatric patients has classically been unfractionated heparin. However, with heparin there are risks of treatment resistance in pediatrics given differences in developmental hemostasis, and contemporary medications such as bivalirudin offer a valuable alternative. Bivalirudin binds to thrombin and has shown encouraging results compared to heparin in pediatric patients receiving ECMO, but no comparison has been made in patients receiving therapeutic anticoagulation. Three years ago, Children’s Mercy Pediatric Hematology department encouraged bivalirudin use over heparin use for therapeutic anticoagulation requiring a continuous infusion. Objectives: Compare clinical and laboratory outcomes in patients receiving …