Hematology Commons^™

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 …

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

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 …

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

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

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

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 …

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 …

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

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.

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 …

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

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 …

Prevalence Of Iron Deficiency In Patients With Inherited Bleeding Disorders, Thomas Cochran, Brian R. Lee, Shannon L. Carpenter

Posters

Background: Patients with bleeding disorders have greater propensity for blood loss and therefore may have a higher prevalence of iron deficiency (ID) when compared to the general population. However, current literature focuses on adolescent females and few studies have assessed the overall prevalence of ID in children with inherited bleeding disorders. Aims: This study aims to identify the prevalence of ID in children with an inherited bleeding disorder. Methods: A retrospective analysis of children with any inherited bleeding disorder seen in Children’s Mercy Hospital’s Hemophilia Treatment Center between 2010 and 2020 was performed. Iron deficiency was defined by recently published …

Functional Evaluation Of A Novel Rpl30 Mutation And Its Role In Diamond Blackfan Anemia (Dba), Alexandra Prosser, Alexandria Cockrell, Danny Miller, Chris Seidel, Tamara Potapova, John M. Perry, Midhat Farooqi, Erin M. Guest, Jennifer Gerton

Research Days

Background: Bone marrow failure is life-threatening and requires prompt, intensive treatment, which is dependent upon identification of genetic drivers. Although there has been investigation into associated genetic mutations, especially in Diamond Blackfan anemia (DBA), the relationship between genotype and phenotype remains unclear.DBA has been identified as a ribosomopathy, also known as a disease associated with defects in ribosome biogenesis. We identified a novel heterozygous variant (c.167+769C >T) in the noncoding region of RPL30 in a patient with clinical diagnosis of DBA. Clinical RNA sequencing (RNA-seq) suggests the variant generates a novel splice acceptor site resulting in truncated RPL30 transcripts. Elucidation …

Increasing Incentive Spirometry Use In Patients With Sickle Cell Disease: Longer Follow-Up Of Pdsa Cycle 1, Alexandra Prosser, Thomas Cochran

Research Days

Problem Statement/Question: Acute chest syndrome (ACS) is one of the leading causes of morbidity and mortality in patients with sickle cell disease. Patients are at higher risk for this complication during hospital admission due to limited mobility leading to decreased lung inflation. The National Heart, Lung, and Blood Institute (NHLBI) recommends providing ten breaths of incentive spirometry (IS) every two hours while awake for patients admitted to the hospital with sickle cell disease to help prevent development of acute chest syndrome. Without this intervention, patients are more likely to develop ACS requiring increased level of care, prolonged hospital stays, and …

Prevalence Of Iron Deficiency In Patients With Inherited Bleeding Disorders, Thomas Cochran, Brian Lee, Shannon Carpenter

Posters

Background: Synthesis of hemoglobin is one of several important roles iron plays in the human body. Approximately 50% of all anemia cases may be caused by iron deficiency which is frequently caused by chronic blood loss. Patients with bleeding disorders have greater propensity for blood loss and therefore may have a higher prevalence of iron deficiency when compared to the general population. However, few studies have assessed the prevalence of iron deficiency in children with inherited bleeding disorders. Objectives: This study aims to identify the prevalence of iron deficiency in children with an inherited bleeding disorder. Methods: A retrospective analysis …

Case Report: Autoimmune Anemia And Thrombocytopenia Following Hematopoietic Stem Cell Transplant, Thomas Cochran, Gabor Oroszi, Nancy Terwilliger, Gabe Metzler, Lejla Music Aplenc

Posters

Background: Autoimmune cytopenias (AIC) post hematopoietic stem cell transplant (HSCT) are rare but exceptionally challenging complications. Here, we report anti E, warm autoimmune hemolytic anemia (WAIHA), cold autoantibody, polyagglutination and immune thrombocytopenia in a 5-year-old boy with a history of chronic granulomatous disease, who underwent an ABO incompatible, mismatched (9/10) unrelated HSCT. Study design: Case report. Four months after HSCT, the patient was admitted for fever, fatigue, and decreased appetite. His original blood type was O+ and donor’s blood type B+. Prior to the admission he was transfused with 2 doses of red cells and 2 doses of platelets. On …

Safety And Efficacy Of Voxelotor In Pediatric Patients With Sickle Cell Disease Aged 4 To 11 Years., Jeremie H. Estepp, Ram Kalpatthi, Gerald Woods, Sara Trompeter, Robert I. Liem, Kacie Sims, Adlette Inati, Baba P D Inusa, Andrew Campbell, Connie Piccone, Miguel R. Abboud, Kim Smith-Whitley, Sandra Dixon, Margaret Tonda, Carla Washington, Noelle M. Griffin, Clark Brown

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Sickle cell disease (SCD) is a devastating, multisystemic disorder that affects millions of people worldwide. The earliest clinical manifestations of SCD can affect infants as young as 6 months of age, and pediatric patients are at risk for acute and life-threatening complications. Early intervention with treatments that target the underlying pathophysiological mechanism of SCD, sickle hemoglobin (HbS) polymerization, are expected to slow disease progression and circumvent disease-associated morbidity and mortality.

PROCEDURE: The HOPE-KIDS 1 trial (NCT02850406) is an ongoing four-part, phase 2a, open-label, single- and multiple-dose study to evaluate the pharmacokinetics, efficacy, and safety of voxelotor-a first-in-class HbS polymerization …

Case Series: Bone Marrow Failure In Teen Siblings With Unique Rps19 Variant, Alexandra Prosser

Research Days

Background: Upon diagnosis of aplastic anemia, inherited bone marrow failure syndromes and acquired etiologies must be considered. Investigating causality is particularly important when multiple family members are affected. It is also essential to identify novel causative genetic variants of bone marrow failure to direct treatment in these patients.

Objectives: To describe the diagnosis and management of siblings who presented two weeks apart with severe pancytopenia and were diagnosed with severe aplastic anemia.

Methods: The first patient is a 13-year-old non-binary female who presented with severe pancytopenia on routine labs. Bone marrow biopsy revealed marked hypocellularity (0-10%) with hypoplasia. The second …

A Novel Approach To Triosephosphate Isomerase Deficiency, Kyra Mccarty

Research Days

Background: Triosephosphate isomerase deficiency (TPID) is a glycolytic pathway enzymopathy causing hemolytic anemia and neurologic dysfunction. TPID is caused by mutations in the TPI1 gene. These patients experience severe hemolytic anemia in infancy followed by progressive neurodegeneration. Diagnosis of TPID is made by measuring decreased TPI enzyme activity elevated dihydroxyacetone phosphate (DHAP) levels in the blood. The diagnosis is confirmed by molecular genetic testing. Neurological involvement typically manifests between 6 and 24 months of age, causing progressive motor dysfunction. Chronic axonal neuropathy is often present on nerve biopsy, and peripheral neuropathy is evident on electrophysiologic studies. Splenectomy and blood transfusions …

Primary Venous Thromboprophylaxis In Pediatric Oncology Patients, Kyra Mccarty

Research Days

Background: The Virchow’s Triad principles of venous stasis, endothelial damage, and hypercoagulability are all features of malignancy and contribute to the increased risk venous thromboembolism (VTE) seen in oncology patients. Additionally, certain chemotherapy treatments, presence of a central venous catheter and immobility are also intrinsically associated with malignancy diagnoses and exacerbate this risk. However, much debate exists over the use of thromboprophylaxis in the pediatric population, primarily due to lack of evidence regarding safety and efficacy, primarily the risk of major bleeding. Multiple retrospective studies have attempted to identify the primary risk factors for VTE in this pediatric oncology patient …

A Study Examining The Safety And Efficacy Of Ferric Carboxymaltose In A Large Pediatric Cohort, Chandni Dargan Md, David Simon Do

Research Days

Background: Iron deficiency anemia (IDA) is common in the pediatric population with varying high-risk factors. Intravenous (IV) iron supplementation has become more desirable in patients with moderate to severe anemia and in patients who are either unresponsive to or have adverse side-effects secondary to oral iron. Iron sucrose and Iron dextran have been traditionally used in pediatrics while ferric carboxymaltose (FCM) has only been FDA approved in adults. One of the major advantages of FCM is the ease of dosing and efficacy. Though FCM was approved for adults in 2013 and there have been no safety concerns, it is not …

A New Paradigm: Ecmo Therapy In Pediatric Hematology/Oncology Patients, Igor Areinamo

Research Days

Background: The use of extracorporeal membrane oxygenation (ECMO) for support of pediatric patients has significantly increased in the past years. Patients with known oncologic diseases, immunodeficiencies, or bone marrow transplants (BMT) have traditionally not been considered candidates for Extracorporeal membrane oxygenation (ECMO) due to perceived high risk for mortality. Ongoing advances have continued to push the boundaries of ECMO use and we report our recent experience with this patient population

Objectives/Goal: To evaluate survival to discharge of pediatric patients who require ECMO support and who also have an underlying hematologic or oncologic disease process. To determine specific patient characteristics that …

Bleeding Disorder Referrals To Hematology Clinic: A Single Institution Experience, Zuri Hudson

Research Days

Background: Our center receives hundreds of referrals yearly for bleeding disorder evaluation both due to bleeding symptoms and secondary to routine preoperative laboratory testing. The evaluation for a bleeding disorder can be challenging due to the wide variability of symptoms as well as the need for accurately interpreting lab results. Bhasin et al showed that 4% of patients referred to hematology based on a preoperative coagulation evaluation had a clinically relevant bleeding disorder. Currently there is little published about the referral patterns to pediatric hematology and the outcomes of these referrals.

Objectives/Goal: To characterize our hematology referrals for bleeding disorder …

Comorbidities And Complications In Adults With Pyruvate Kinase Deficiency., Audra N. Boscoe, Yan Yan, Elizabeth Hedgeman, Eduard J. Van Beers, Hanny Al-Samkari, Wilma Barcellini, Stefan W. Eber, Bertil Glader, Hassan M. Yaish, Satheesh Chonat, Mukta Sharma, Kevin H M Kuo, Ellis J. Neufeld, Heng Wang, Madeleine Verhovsek, Sujit Sheth, Rachael F. Grace

Manuscripts, Articles, Book Chapters and Other Papers

OBJECTIVES: Pyruvate kinase (PK) deficiency is caused by PKLR gene mutations, leading to defective red blood cell glycolysis and hemolytic anemia. Rates of comorbidities and complications by transfusion history and relative to the general population remain poorly quantified.

METHODS: Data for patients aged ≥ 18 years with two confirmed PKLR mutations were obtained from the PK deficiency Natural History Study (NCT02053480). Frequencies of select conditions were compared with an age- and sex-matched cohort from a general insured US population without PK deficiency.

RESULTS: Compared with the matched population (n = 1220), patients with PK deficiency (n = 122) had significantly …

Tisagenlecleucel Infusion In Patients With Relapsed/Refractory All And Concurrent Serious Infection., Erin Hall, Dwight E. Yin, Rakesh K. Goyal, Atif Ahmed, Grace S. Mitchell, Shawn D. St Peter, Terrie Flatt, Ibrahim A. Ahmed, Weijie Li, Richard J. Hendrickson, Keith August, Douglas Myers

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Tisagenlecleucel, an anti-CD19 chimeric antigen receptor T (CAR-T) cell therapy, has demonstrated durable efficacy and a manageable safety profile in pediatric and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL) in the ELIANA pivotal trial and real-world experience. Experience from investigator-led studies prior to ELIANA suggests that infections and inflammatory conditions may exacerbate the severity of cytokine release syndrome (CRS) associated with CAR-T cell therapy, leading to extreme caution and strong restrictions for on-study and commercial infusion of tisagenlecleucel in patients with active infection. CRS intervention with interleukin (IL)-6 blockade and/or steroid therapy was introduced late in …

Pre-Transplant Marital Status And Hematopoietic Cell Transplantation Outcomes., J Tay, S Beattie, C Bredeson, R Brazauskas, N He, Ibrahim A. Ahmed, M Aljurf, M Askar, Y Atsuta, S Badawy, A Barata, A M Beitinjaneh, N S Bhatt, D Buchbinder, J Cerny, S Ciurea, A D'Souza, J Dalal, N Farhadfar, C O Freytes, S Ganguly, U Gergis, S Gerull, H M Lazarus, T Hahn, S Hong, Y Inamoto, N Khera, T Kindwall-Keller, R T Kamble, J M Knight, Y N Koleva, A Kumar, J Kwok, H S Murthy, R F Olsson, M Angel Diaz-Perez, D Rizzieri, S Seo, S Chhabra, H Schoemans, H C Schouten, A Steinberg, K M Sullivan, J Szer, D Szwajcer, M L Ulrickson, L F Verdonck, B Wirk, W A Wood, J A Yared, W Saber

Manuscripts, Articles, Book Chapters and Other Papers

Background: Evidence about the impact of marital status before hematopoietic cell transplantation (hct) on outcomes after hct is conflicting.

Methods: We identified patients 40 years of age and older within the Center for International Blood and Marrow Transplant Research registry who underwent hct between January 2008 and December 2015. Marital status before hct was declared as one of: married or living with a partner, single (never married), separated or divorced, and widowed. We performed a multivariable analysis to determine the association of marital status with outcomes after hct.

Results: We identified 10,226 allogeneic and 5714 autologous hct cases with, respectively, …

Active Music Engagement And Cortisol As An Acute Stress Biomarker In Young Hematopoietic Stem Cell Transplant Patients And Caregivers: Results Of A Single Case Design Pilot Study., Steven J. Holochwost, Sheri L. Robb, Amanda K. Henley, Kristin Stegenga, Susan M. Perkins, Kristen A. Russ, Seethal A. Jacob, David Delgado, Joan E. Haase, Caitlin M. Krater

Manuscripts, Articles, Book Chapters and Other Papers

This paper reports the results of a single case design pilot study of a music therapy intervention [the Active Music Engagement (AME)] for young children (age 3.51 to 4.53 years) undergoing hematopoietic stem cell transplantation (HCST) and their caregivers. The primary aims of the study were to determine feasibility/acceptability of the AME intervention protocol and data collection in the context of HCST. Secondary aims were to examine caregivers' perceptions of the benefit of AME and whether there were changes in child and caregiver cortisol levels relative to the AME intervention. Results indicated that the AME could be implemented in this …

The Spectrum And Severity Of Bleeding In Adolescents With Low Von Willebrand Factor-Associated Heavy Menstrual Bleeding., Lakshmi Srivaths, Charles G. Minard, Sarah H. O'Brien, Allison P Wheeler, Eric Mullins, Mukta Sharma, Robert Sidonio, Shilpa Jain, Ayesha Zia, Margaret V. Ragni, Roshni Kulkarni, Jennifer E. Dietrich, Peter A. Kouides

Manuscripts, Articles, Book Chapters and Other Papers

Low von Willebrand factor (VWF) in adults is associated with significant bleeding, most notably heavy menstrual bleeding (HMB) and postpartum hemorrhage, although this has not been characterized in adolescents. The objectives of this analysis of a multicenter single arm observational cohort study in adolescents with low VWF-associated HMB were to describe the bleeding phenotype, HMB severity, and related complications. Eligibility criteria included postmenarchal females(Pictorial Blood Assessment Chart [PBAC] score >100) and low VWF (2 values of VWF activity ≥30 and ≤50 IU/dL). Patients diagnosed with other bleeding disorders were ineligible. Clinical phenotype data, including PBAC and Bleeding Assessment Tool (BAT) …