Dr. Abdulmalik’s research interests broadly encompass the hemoglobinopathies and red blood cell disorders, with particular focus on sickle cell disease and beta-thalassemia. Though both disorders are consequences of simple genetic mutations, the implications of these mutations are inherently complex, and include significant morbidities, lifelong challenges, reduced quality of life, and early mortality in patients; as well as substantial healthcare burden on families, caregivers, and society in general. Dr. Abdulmalik’s current research focuses on three modalities of countering disease pathophysiology. The first approach, in tandem with a multidisciplinary team of collaborators, utilizes medicinal chemistry and structural biology to design novel drugs that block the primary pathophysiology of sickle cell disease, i.e., the polymerization of sickle hemoglobin.   Representative Publications: Design, Synthesis, and Biological Evaluation of Ester and Ether Derivatives of Antisickling Agent 5-HMF for the Treatment of Sickle Cell Disease New developments in anti-sickling agents: can drugs directly prevent the polymerization of sickle haemoglobin in vivo? Forced chromatin looping raises fetal hemoglobin in adult sickle cells to higher levels than pharmacologic inducers Clinical Implications of the Association of Fetal Hemoglobin with Peripheral Oxygen Saturation in Sickle Cell Disease Crystal structure of carbonmonoxy sickle hemoglobin in R-state conformation Online profile

Dr. Carlton Dampier is a pediatric hematologist-oncologist in Atlanta, Georgia and is affiliated with multiple hospitals in the area, including Children's Healthcare of Atlanta and Grady Memorial Hospital. He received his medical degree from University of Chicago Pritzker School of Medicine and has been in practice for more than 20 years. His broad research interest is the assessment of patient-reported outcomes and subsequent symptom management in children and adolescents, particularly those with pain from sickle cell disease. He has led or participated in a number of studies validating specific assessment tools, as well as longitudinal observation studies examining symptom frequency by self-report or parent-proxy reporting. He has led or participated in a number of clinical trials of analgesics or investigational drugs for the treatment of vaso-occlusive sickle cell pain. As a research administrator, he has a broad interest in process improvement and quality management of clinical trials, as well as research ethics and scientific integrity in the conduct of clinical trials.   Representative Publications: IMPROVE trial: a randomized controlled trial of patient-controlled analgesia for sickle cell painful episodes: rationale, design challenges, initial experience, and recommendations for future studies Opioid patient controlled analgesia use during the initial experience with the IMPROVE PCA trial: a phase III analgesic trial for hospitalized sickle cell patients with painful episodes Red blood cell alloimmunization in sickle cell disease: prevalence in 2010 Tissue factor-positive monocytes in children with sickle cell disease: correlation with biomarkers of haemolysis Inpatient management of sickle cell pain: a 'snapshot' of current practice Definitions of the phenotypic manifestations of sickle cell disease Online profile

Yvonne M. Carroll, RN, JD is the Director of Patient Services in the Department of Hematology at St. Jude Children’s Research Hospital.  Yvonne graduated with a Bachelors of Nursing Degree from the University of Tennessee and a Law degree from the University of Cincinnati. She joined SJCRH in 1999 in the Department of Hematology and served in several positions before becoming the Director of Patient Services. Yvonne is active in Hematology and the sickle cell disease community and serves on the Governor’s Genetic Advisory Committee for the State of TN. Throughout the years, Yvonne has been a Principal Investigator and Program Manager on several sickle cell grants and has spoken in numerous venues on sickle cell disease. Yvonne has developed partnerships with several community organizations and rural healthcare workers to improve awareness and treatment for people with sickle cell disease. She serves as president of the International Association of Sickle Cell Nurses and Professional Associates (IASCNAPA) which recently became partners with the Sickle Cell Community Consortium and the Sickle Cell Disease Coalition.   Representative Publications: Caregiver Perspectives of Stigma Associated With Sickle Cell Disease in Adolescents Genetic education and sickle cell disease: feasibility and efficacy of a program tailored to adolescents Therapy preference and decision-making among patients with severe sickle cell anemia and their families Online profile

Dr. Campbell's distinguished training and career path began at Morehouse College. He continued medical school at Case Western Reserve University and completed post graduate training at Massachusetts General Hospital (Harvard) and Lurie Children's Hospital (Northwestern University). Prior to joining Children’s National, he was Director of the Comprehensive Sickle Cell Center at the University of Michigan, a position he held since 2005. His research interests span several topics in sickle cell disease including pulmonary complications, fetal hemoglobin switching in transgenic sickle cell mice, phenotype/genotype relationships, and renal complications.   Areas of Interest: Pulmonary Hypertension in sickle cell disease Phenotype/genotype Studies within twin and siblings with sickle cell disease Morbidity and mortality in transitioning sickle cell patients Fetal hemoglobin switching within sickle cell trangenic mice   Representative Publications: Differences in the clinical and genotypic presentation of sickle cell disease around the world An analysis of inpatient pediatric sickle cell disease: Incidence, costs, and outcomes mTOR Inhibition improves anaemia and reduces organ damage in a murine model of sickle cell disease Protective effect of HLA-DQB1 alleles against alloimmunization in patients with sickle cell disease Single-session biofeedback-assisted relaxation training in children with sickle cell disease Hereditary Persistence of Fetal Hemoglobin Caused by Single Nucleotide Promoter Mutations in Sickle Cell Trait and Hb SC Disease Online profile

Andrea Williams founded and leads the Children’s Sickle Cell Foundation in Pittsburgh, PA. She is a parent representative and site lead in the project. Her youngest son, Jonathan, was diagnosed at birth with sickle cell disease (SCD) through the universal National Newborn Screening Program. Her son’s diagnosis spurred Andrea to start the Children’s Sickle Cell Foundation (CSCF) in Pittsburgh. She envisioned an organization that would specifically serve the needs of children with SCD and their families and that would help fund research for a cure. Jonathan’s special needs presented unique challenges that changed her involvement from helping out at school events to focusing on educational advocacy for her son, and for children like him. The situation with Jonathan made Andrea realize that what happened in her family could be happening in other families as well. The steps she took to support her son needed to be discussed at a systems level so that many other children and families would benefit. Andrea was learning a lot as a parent partner and site lead for the Sickle Cell Disease Treatment Demonstration Project, a component of NICHQ’s WISCH initiative. That was her first experience with using quality improvement methods to improve existing systems, and she chose to participate because of the potential for making real changes in the care and treatment of people with sickle cell disease. Today, Andrea advocates children with Sickle Cell Disease and their families.   Representative Publications: Sickle Cell Disease, 2015: A Patient Advocate's Perspective Online profile

Mandy David is a senior health communications specialist and a deputy project director at AIR. She is a certified physician assistant (PA), with specific training in sickle cell disease management. Additionally, she has been trained in addiction medicine, primary care and cardiac and basic life support. David has worked with pediatric, adult and geriatric patients, and has seven years’ experience working with sickle cell patients. Before joining AIR, David served as a program and clinical manager at the Johns Hopkins University Sickle Cell Infusion Center, as well as a primary care PA at ULNACS Primary Care, and a neurosurgical and critical care PA at Georgetown University Hospital, among other PA positions. She is currently working toward her master of public health degree at the Johns Hopkins Bloomberg School of Public Health.   Representative Publications: Impact of a dedicated infusion clinic for acute management of adults with sickle cell pain crisis Improvements in haemolysis and indicators of erythrocyte survival do not correlate with acute vaso-occlusive crises in patients with sickle cell disease: a phase III randomized, placebo-controlled, double-blind study of the Gardos channel blocker senicapoc (ICA-17043) Online profile

Dr. Rivers is the Director at Sickle Cell Transition Adolescent-Adult Readiness Clinic (STAR) program. Her research interests focus on the Adeno Associated Virus as a vector for gene therapy in sickle cell disease. She has received NIH K08 funding for lab research inducers of fetal hemoglobin. The research (K01 HL103172-01, NIH/ NIHLBI) led by Dr. Rivers focuses on AAV capsid engineering to facilitate expression of therapeutic levels of beta-globin in mouse hematopoietic stem cells. Because everyone is not a candidate for transplantation, successful treatment for sickle cell disease needs to include the development of therapeutic options beside hydroxyurea. Under the mentorship of Dr. Don Lavelle and Dr. Joseph De Simone at the University of Illinois Chicago, the research of Dr. Rivers also focuses on the possible phenotypic correction of sickle disease by induction of hemoglobin F with the combination treatment of Decitabine and novel drugs.   Representative Publications: Adverse Reactions to Pneumococcal Vaccine in Pediatric and Adolescent Patients with Sickle Cell Disease Pharmacological inhibition of LSD1 and mTOR reduces mitochondrial retention and associated ROS levels in the red blood cells of sickle cell disease The LSD1 inhibitor RN-1 induces fetal hemoglobin synthesis and reduces disease pathology in sickle cell mice RN-1, a potent and selective lysine-specific demethylase 1 inhibitor, increases γ-globin expression, F reticulocytes, and F cells in a sickle cell disease mouse model Drug-loaded sickle cells programmed ex vivo for delayed hemolysis target hypoxic tumor microvessels and augment tumor drug delivery Online profile

Nancy Green, MD is a pediatric hematologist with expertise in the diagnosis and treatment of the full-range of pediatric blood disorders, with a special interest in hemoglobinopathies such as sickle cell disease. Her interest in sickle cell disease began while in college, where Dr. Green was attracted to its clinical complexity, laboratory features, and research opportunities to improve the health of people with the disease. Her current research focuses on optimizing treatments for sickle cell disease, especially the drug hydroxyurea – the primary therapy for patients. She is studying the differences among patients in their response to this drug: the genetics of response to hydroxyurea, adherence to therapy, and cultural and other aspects. She is also studying the hematologic aspects of bone marrow transplantation, which can cure sickle cell disease but is not an option for many patients. Dr. Green hopes to contribute to the field by enhancing the understanding of how patients with sickle cell disease respond to hydroxyurea, with the goal of clarifying how it can be most effectively used and by whom. For families whose children do not respond well to this drug, she hopes her work will help them as they think about their options for treatment. Dr. Green's research also focuses on newborn screening, a public health program in which infants are tested for a variety of disorders usually before the baby goes home from the newborn nursery.   Representative Publications: Study protocol for a randomized controlled trial to assess the feasibility of an open label intervention to improve hydroxyurea adherence in youth with sickle cell disease Community Health Workers as Support for Sickle Cell Care OCT-Angiography and Ultra-Widefield Fluorescein Angiography for Early Detection of Adolescent Sickle Retinopathy New Ways to Detect Pediatric Sickle Cell Retinopathy: A Comprehensive Review Randomized feasibility trial to improve hydroxyurea adherence in youth ages 10-18 years through community health workers: The HABIT study Enhanced Long-Term Brain Magnetic Resonance Imaging Evaluation of Children with Sickle Cell Disease after Hematopoietic Cell Transplantation Online profile

Dr. Krishnamurti graduated from the Armed Forces Medical College, University of Pune, Pune, Maharashtra, India in 1980. He works in Atlanta, GA and specializes in Pediatric Hematology-Oncology. Dr. Krishnamurti is affiliated with Children's Healthcare of Atlanta Egleston Campus and Children's Heathcare Of Atlanta At Scottish Rite. He is also the Chair of Blood and Marrow Transplant at Children’s Healthcare of Atlanta/Emory University.   Representative Publications: Haematopoietic stem cell transplantation for sickle cell disease - current practice and new approaches Hospitalization for pain in patients with sickle cell disease treated with sildenafil for elevated TRV and low exercise capacity IMPROVE trial: a randomized controlled trial of patient-controlled analgesia for sickle cell painful episodes: rationale, design challenges, initial experience, and recommendations for future studies Opioid patient controlled analgesia use during the initial experience with the IMPROVE PCA trial: a phase III analgesic trial for hospitalized sickle cell patients with painful episodes Nitric oxide for inhalation in the acute treatment of sickle cell pain crisis: a randomized controlled trial Clinical risks and healthcare utilization of haematopoietic cell transplantation for sickle cell disease in the U.S. using merged databases Proponent or collaborative: Physician perspectives and approaches to disease modifying therapies in sickle cell disease Online profile

Dr. Fields practices in the Division of Hematology and Oncology at St. Louis Children's Hospital. She is also affiliated with Barnes-Jewish Hospital. She delivers Hematology Services, Oncology Services, and Sickle Cell Disease Program.   Specialties: -Oncology-Pediatric - certified -Hematology-Pediatric - certified -Pediatrics - certified     Representative Publications: Large-Vessel Vasculopathy in Children With Sickle Cell Disease: A Magnetic Resonance Imaging Study of Infarct Topography and Focal Atrophy A pilot study of parent education intervention improves early childhood development among toddlers with sickle cell disease Red blood cell storage duration is not associated with clinical outcomes for acute chest syndrome in children with sickle cell disease Higher-than-expected prevalence of silent cerebral infarcts in children with hemoglobin SC disease Online profile