AMANDA PFEIFFER

ACTIVE STUDIES

Cardiac Magnetic Resonance Imaging to Identify Diffuse Myocardial Fibrosis in Sickle Cell Disease and Other Hemolytic Anemias

• March 2022 to Present - Lead Data Coordinator
• Study Objective: Frequency and severity of diffuse myocardial fibrosis in non-HbSS (non-SCA) forms of SCD. Estimate the frequency and severity of diffuse myocardial fibrosis, quantified by ECV, in a representative sample of children and adults with sickle-hemoglobin C disease (HbSC) and sickle-Beta+thalassemia (HbSB+), two common forms of SCD in which assessments for diffuse myocardial fibrosis have not yet been performed.

Prospective Identification of Variables as Outcomes for Treatment (PIVOT): A Phase II clinical trial of hydroxyurea for children and adults with HbSC disease

• August 2021 to Present - Lead Data Coordinator
• Study Objective: 1. To measure the toxicities of hydroxyurea treatment on laboratory parameters. 2. To assess the effects of hydroxyurea treatment on a variety of sickle-related clinical and laboratory parameters in a large cohort of children and adults with HbSC disease. 3. To identify which study endpoints are suitable for a future Phase III trial of patients with HbSC disease receiving hydroxyurea therapy.

Alternative Dosing And Prevention of Transfusions (ADAPT)

• August 2021 to Present - Lead Study Monitor
• Study Objective: To compare the rates of blood transfusions overall and by specific indications in children with sickle cell anaemia (SCA), prior to and during hydroxyurea treatment

Losartan for Diffuse Myocardial Fibrosis in Sickle Cell Disease: A prospective, phase II study

• May 2021 to Present - Lead Research Coordinator
• Study Objective: To assess the efficacy of losartan in stabilizing or reducing diffuse myocardial fibrosis in SCD. Our primary endpoint is a stability or reduction in ECV from baseline at 1 year. To assess the efficacy of losartan in improving diastolic function, exercise capacity, and the CMR-based structural and functional measures.

Management of Care Transitions for Emerging Adults with Sickle Cell Disease (COMETS)

• July 2021 to Present - Lead Research Coordinator
• Study Objective: The purpose of the study is to determine the comparative effectiveness of CHW programs and mHealth among emerging adults with SCD during transition versus enhanced usual care to improve health-related quality of life and acute care use for transitioning youth with SCD.

Prospective Multi-Center Evaluation of the Duration of Therapy for Thrombosis in Children (the "Kids-DOTT" Trial)

• 2019 to Present - Lead Research Coordinator
• Study Objective: To evaluate the efficacy of short-duration (6 weeks total) versus conventional-duration (3 months total) anticoagulation for first-episode acute venous thrombosis among children in whom thrombus resolution is evident after the initial 6 weeks of anticoagulant therapy

Phase 3, prospective, multicenter, open-label study to investigate safety, immunogenicity, and hemostatic efficacy of PEGylated Factor VIII (BAX 855) in previously untreated patients (PUPs) and minimally treated patients (MTPs

• 2019 to Present
• Role: Lead Research Coordinator
• Study Objective: The primary objective is to determine safety including immunogenicity of BAX 855 based on the incidence of inhibitor development to FVIII (? 0.6 BU/mL using the Nijmegen modification of the Bethesda assay)

Hydroxyurea Optimization through Precision Study (HOPS): A prospective, multi-center, randomized trial of personalized, pharmacokinetics (PK)-guided dosing of hydroxyurea versus standard weight-based dosing for children with sickle cell anemia

• 2017 to Present - Project Manager, Lead Site Coordinator
• Study Objective: To compare a novel individualized hydroxyurea dosing approach, using sparse PK sampling and pharmacometrics-based modeling, to standard conservative step-wise dose escalation for children with SCA. Whole blood samples will be collected after an initial fixed hydroxyurea dose and the total hydroxyurea exposure will be estimated using a population PK model to predict the optimal starting dose for each study participant. Children will be randomized to either the standard 20 mg/kg daily dose (Standard Arm) or the predicted PK-based dose (Alternative Arm), with subsequent dose reductions and escalations, using the same rules for each arm, based on scheduled peripheral blood counts.

Therapeutic Response Evaluation and Adherence Trial (2014 to Present, Lead Research Coordinator)

• 2014 to Present - Lead Research Coordinator
• Study Objective: To reduce the time required to reach maximum tolerated dose (MTD) of hydroxyurea for children with sickle cell anemia (SCA) using a novel pharmacometrics model

CLOSED STUDIES

Assessment of Neurocognitive Status With Education through Research in Sickle Cell Disease (ANSWERS)

• 2020 to 2022 - Lead Research Coordinator
• Study Objective: To determine the prevalence of school service utilization (specifically Individualized Education Plan (IEP) and 504 Plans) in patients with all genotypes of sickle cell disease who have historically undergone the Brief School Needs Inventory (BSNI) from 2016 present. To evaluate the neurocognitive performance of patients with specifically sickle cell anemia (SCA, genotypes HbSS and HbS-ß0thalassemia) who initiated hydroxyurea at a young age (25%).

A phase 4, open-label, single-center study to assess pharmacokinetic characteristics and safety of Endari in patients with sickle cell disease (EM-PK-01)

• October 2020 to July 2021 - Lead Research Coordinator
• Study Objective: To investigate the relationship between oral administration of Endari (L-glutamine oral powder) and L-glutamine pharmacokinetics (PK), mainly describing drug absorption, metabolism and elimination, food-drug interactions and impact of weight and age on pharmacokinetics with dose escalation in two different age cohorts of sickle cell disease (SCD) patients and 1 cohort of healthy adult volunteers. Additionally, to report treatment related adverse events (TRAEs), dose-limiting toxicities associated with ammonia and glutamate levels, and drug adherence.

Serology to COVID for Recording Exposure and Evaluating Needs (SCREEN)

• 2020 to 2021 - Data Coordinator, Research Coordinator
• Study Objective: The purpose of this research study is 1) to conduct a prospective longitudinal surveillance research study, enrolling up to 230 residents and fellows, and then following their clinical and laboratory parameters for up to 24 months; and 2) to support the ongoing development of diagnostic serological techniques for COVID-19 infection. The overall goal is to investigate the seroprevalence of SARS-COV-2 infection over time, and to better understand factors associated with the acquisition of SARS-COV-2 infection among pediatric trainees at CCHMC.

Repeated Employee Testing for Understanding our Return to Normal (RETURN)

• 2020 to 2021 - Data Coordinator, Research Coordinator
• Study Objective: The purpose of this research study is 1) to conduct a prospective longitudinal surveillance research trial, enrolling up to 200 CCHMC employees as they come back to work, and then following their clinical and laboratory parameters for up to 12 months; and 2) to support the ongoing development of diagnostic techniques for COVID-19. The overall goal is to investigate patterns of SARS-COV-2 infection, including immunological recovery and genetic risk factors, among our employees to better understand how to safely reintroduce the CCHMC work force back into their normal routines.

Gene Transfer in Patients with Sickle Cell Disease Using a Gamma Globin Lentivirus Vector: An Open Label Phase I/II Pilot Study (November 2019 to December 2019, Data Entry Assistant)

• 2019 - Data Entry Assistant
• Study Objective: Evaluate the safety of: Bone marrow collection from subjects with SCD, Gene transfer of sGbG vector into sickle CD34+ cells, Chemotherapy conditioning with melphalan

Evaluation of aspirin efficacy in post-procedural pediatric patients in non-cardiac settings

• 2018 to 2021 - Lead Research Coordinator
• Study Objective: The purpose of this study is to explore the reliability of aspirin monitoring in the post-procedural setting for pediatric patients who are not undergoing cardiac procedures.

Dissemination and Implementation of Stroke Prevention Looking at the Care Environment (DISPLACE)

• 2018 to 2020 - Lead Research Coordinator
• Study Objective: Use case records from 2010 to 2016; we aim to determine the rate of annual TCD screening and initiation of Chronic Red Blood Cell Transfusion therapy (CRCT) per STOP protocol among all patients associated with consortium institutions.

Natural history study of factor IX treatment and complications (B– Natural)

• 2018 - Lead Research Coordinator
• Study Objective: Document treatment-related characteristics of patients with FIX deficiency, Examine the concordance of clinical characteristics within affected brother pairs/groups with FIX deficiency, Screen for indicators or renal disease, Describe patient-and treatment-related risk factors for the development of inhibitory and non-inhibitory antibodies toward FIX, Examine the clinical management of patients with inhibitory antibodies, the use of immune tolerance induction, ITI outcome and associated sequelae, Conduct a health economic assessment of the management of FIX deficiency

Studying Medication Education and Adherence with Red blood cell smears in Sickle Cell Anemia

• 2017 to 2021 - Sub-Investigator
• Study Objective: To determine the optimal patient-friendly way in which to share laboratory data and blood smear images. During the pilot phase of the study, we will use a variety of methods (printouts, interactive tablets, text messages/emails) to share laboratory trends and blood smear images with patients and families. Through direct patient and family feedback, we aim to determine which method is most effective and well-received by the patients and their families. This will allow for a more effective interventional phase of the study.

Sickle Cell Disease and Growth Retrospective Study (2015 to 2018, Lead Research Coordinator)

• 2015 to 2018 - Lead Research Coordinator
• Study Objective: The purpose of this study is to investigate the growth, thyroid status, pubertal status, and bone health of children with sickle cell disease and to determine the effect of hydroxyurea on endocrine function, including growth and growth velocity.

Children's for Children's Parent Advisory Committee (July 2020 to Present)

Participates in discussions for ideas and solutions to make the organization a stronger support for the children, teachers, and parents (whom at least one, is a CCHMC employee). Topics include COVID masking and restrictions, kindergarten preparation, teacher-parent communications, student development and diversity and inclusion of the educational elements to all the classrooms.

CRC Advancement Coordinator (June 2019 to Present)

Responsible for coordinating the CRC Advancement applications as a permanent member of the CRC Advancement Committee. This includes training volunteering reviewers, notifying application assignments, ensuring all applications meet the minimum criteria for advancement. This role requires scheduling all monthly board meetings, agendas and reports for the full CRC Advancement Review Board.

Sickle Cell Research Day (2014 to 2019)

Assisted in organizing annual event including calling all patients for RSVPs, patient participation in Hydroxyurea panel and family activities and enrollment to research studies.

Facilitator Informed Consent Education Course (2019 to Present)

Assists to educate new Clinical Research Professionals at CCHMC about the Informed Consent Process for research processes in the safe environment.

Licensed Professional Counselor

State of Ohio- Number: C.1400341

Expires: September 22, 2023

SOCRA Clinical Research Professional Certification

Expires: May 1, 2023

• CCHMC Mentoring Program- 2020 to 2021
• CMRSC Wellness Committee– 2015 to 2017
• American Counseling Association– 2015 to Present

• CITI Certified November 11, 2013
  Recertified December 2021
• Child Psychology Grand Rounds
• Schwartz Center Grand Rounds
• Hematology Grand Rounds
• Clinical Research Professionals Monthly Meeting
• Core Clinical Research Training
  May 5-8, 2016

• Karkoska K, Pfeiffer A, Beebe DW, Quinn CT, Niss O, McGann PT. Early hydroxyurea use is neuroprotective in children with sickle cell anemia. Am J Hematol. 2022 Oct;97(10):E368-E370. doi: 10.1002/ajh.26664. Epub 2022 Jul 26. PMID: 35836401.
• Quinn, C.T., Niss, O., Dong, M., Pfeiffer, A., Korpik, J., Reynaud, M., Bonar, H., Kalfa, T.A., Smart, L.R., Malik, P., Ware, R.E., Vinks, A.A. and McGann, P.T. (2021), Early initiation of hydroxyurea (hydroxycarbamide) using individualised, pharmacokinetics-guided dosing can produce sustained and nearly pancellular expression of fetal haemoglobin in children with sickle cell anaemia. Br. J. Haematol., 194: 617-625. https://doi.org/10.1111/bjh.17663
• Sadaf A, Quinn CT, Korpik JB, Pfeiffer A, Reynaud M, Niss O, Malik P, Ware RE, Kalfa TA, McGann PT. Rapid and automated quantitation of dense red blood cells: A robust biomarker of hydroxyurea treatment response. Blood Cells Mol Dis. 2021 May 11;90:102576. doi: 10.1016/j.bcmd.2021.102576. Epub ahead of print. PMID: 34020272.
• Karkoska, K, Quinn, CT, Niss, O, et al. Hydroyxurea improves cerebral oxygen saturation in children with sickle cell anemia. Am J Hematol. 2021; 96: 538– 544. https://doi.org/10.1002/ajh.26120
• Perez-Plazola, M.S., Tyburski, E.A., Smart, L.R. et al. AnemoCheck-LRS: an optimized, color-based point-of-care test to identify severe anemia in limited-resource settings. BMC Med 18, 337 (2020). https://doi.org/10.1186/s12916-020-01793-6
• Meier, E.R., Creary, S.E., Heeney, M.M. et al. Hydroxyurea Optimization through Precision Study (HOPS): study protocol for a randomized, multicenter trial in children with sickle cell anemia. Trials 21, 983 (2020). https://doi.org/10.1186/s13063-020-04912-z
• Ronay Thomas, Patrick T. McGann, Andrew Beck, Amanda Pfeiffer, Kyesha M James; Characterization of Community-Based Socioeconomic Factors, Utilization, and Adherence in Children with Sickle Cell Disease. Blood 2019; 134 (Supplement_1): 4686. doi: https://doi.org/10.1182/blood-2019-130637
• Charles T. Quinn, Omar Niss, Amanda Pfeiffer, Jennifer Korpik, Holly Bonar, Mary Reynaud, Russell E. Ware, Patrick T. McGann; Pharmacokinetics-Guided Dosing of Hydroxyurea Can Achieve Near-Pancellular Fetal Hemoglobin Expression in Sickle Cell Anemia: F-Cell Analysis As a Benchmark for Disease-Modifying Therapy. Blood 2019; 134 (Supplement_1): 892. doi: https://doi.org/10.1182/blood-2019-130414
• Boucher, Alex; Pfeiffer, Amanda; Bedel, Ashley; Young, Jennifer; McGann, Patrick T. / Utilization trends and safety of intravenous iron replacement in pediatric specialty care: A large retrospective cohort study. In Pediatric Blood and Cancer. 2018; Vol. 65, No. 6. https://doi.org/10.1002/pbc.26995