Rachael Grace, MD, MMSc

Director, Hematology Clinical Research
Associate Professor of Pediatrics, Harvard Medical School
Image
Rachael Grace, MD, MMSc

Rachael Grace, MD, MMSc

Director, Hematology Clinical Research
Associate Professor of Pediatrics, Harvard Medical School
Education
Graduate School
MMSc
Harvard Medical School
Boston
MA
Medical School
Brown University
2003
Providence
RI
Internship
Pediatrics
Boston Children's Hospital/Boston Medical Center
2004
Boston
MA
Residency
Pediatrics
Boston Combined Residency Program (BCRP)
2006
Boston
MA
Fellowship
Pediatric Hematology-Oncology
Boston Children's Hospital/ Dana-Farber Cancer Institute
2010
Boston
MA
Certifications
American Board of Pediatrics (General)
American Board of Pediatrics (Hematology-Oncology)
Professional History

 Rachael Grace, MD, MMSc, is a pediatric hematologist and clinical researcher at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. She is the medical director of both the Hematology Ambulatory Program and the Hematology Clinical Research Program. Dr. Grace enjoys clinical care of pediatric patients and her research interests are focused on improving outcomes in the care and treatment of individuals with immune cytopenias.

Dr. Grace is the lead investigator for the Pyruvate Kinase Deficiency Natural History Study, a global registry for patients with PK deficiency. She is a site investigator for the DRIVE-PK and ACTIVATE mitapivat (AG-348) clinical trials and the PEAK registry. She is also involved in multiple clinical research studies in immune cytopenias and is the director of the coordinating center for the ITP Consortium of North America (ICON), a research network of pediatric idiopathic thrombocytopenic purpura investigators from over 50 institutions in the U.S. and Canada.

Dr. Grace is an Associate Professor of Pediatrics at Harvard Medical School. She received her MD at Brown University and received her master’s degree in clinical research at Harvard Medical School. Dr. Grace completed her residency at Boston Children’s Hospital and completed her pediatric hematology/oncology fellowship at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center.

Publications

Treatment Landscape in Pediatric Immune Thrombocytopenia: Addressing Unmet Needs. View Abstract
Critical Bleeding in Adults and Children with Immune Thrombocytopenia: A Multicenter Cohort Study. View Abstract
Working toward international consensus defining pediatric "refractory ITP". View Abstract
Real-world insights from a cohort of approximately 2000 individuals who were analysed using a freely available next-generation sequencing anaemia screening programme. View Abstract
Correlation of Genetic Mutation With Outcomes in Children With Hereditary Spherocytosis Undergoing Partial Splenectomy: A Multicentre Study. View Abstract
T cell and autoantibody profiling for primary immune regulatory disorders. View Abstract
Treatment of Critical Bleeds in Patients With Immune Thrombocytopenia: A Systematic Review. View Abstract
Genetic variants in canonical Wnt signaling pathway associated with pediatric immune thrombocytopenia. View Abstract
What is in a name: defining pediatric refractory ITP. View Abstract
Quality of life in childhood immune thrombocytopenia: Revision of the Kids' ITP Tools (KIT). View Abstract
The 2022 review of the 2019 American Society of Hematology guidelines on immune thrombocytopenia. View Abstract
Comorbidities and complications in adult and paediatric patients with pyruvate kinase deficiency: Analysis from the Peak Registry. View Abstract
Mitapivat improves ineffective erythropoiesis and iron overload in adult patients with pyruvate kinase deficiency. View Abstract
Clinically meaningful improvements in patient-reported outcomes in mitapivat-treated patients with pyruvate kinase deficiency. View Abstract
Bone mineral density in adult patients with pyruvate kinase deficiency on long-term mitapivat treatment. View Abstract
Diagnosis and management of pyruvate kinase deficiency: international expert guidelines. View Abstract
Pyruvate kinase activators for treatment of pyruvate kinase deficiency. View Abstract
Psychometric validation of the Pyruvate Kinase Deficiency Diary and Pyruvate Kinase Deficiency Impact Assessment in adults in the phase 3 ACTIVATE trial. View Abstract
HLA antibodies in fetal and neonatal alloimmune thrombocytopenia. View Abstract
The Pyruvate Kinase Deficiency Global Longitudinal (Peak) Registry: rationale and study design. View Abstract
Updates and advances in pyruvate kinase deficiency. View Abstract
Early-onset reduced bone mineral density in patients with pyruvate kinase deficiency. View Abstract
Pediatric refractory immune thrombocytopenia: A systematic review. View Abstract
Long-term risk of developing immune thrombocytopenia and hematologic neoplasia in adults with mild thrombocytopenia. View Abstract
Utilization of an ITP quality improvement pathway improves adherence to management guidelines. View Abstract
An update on pediatric ITP: differentiating primary ITP, IPD, and PID. View Abstract
SARS-CoV-2 vaccination in pediatric patients with immune thrombocytopenia. View Abstract
Diagnosis, monitoring, and management of pyruvate kinase deficiency in children. View Abstract
Mitapivat versus Placebo for Pyruvate Kinase Deficiency. View Abstract
Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency. View Abstract
Thrombopoietin receptor agonists and rituximab for treatment of pediatric immune thrombocytopenia: A systematic review and meta-analysis of prospective clinical trials. View Abstract
Characteristics and outcomes of autoimmune hemolytic anemia after pediatric allogeneic stem cell transplant. View Abstract
Phase 3 randomised trial of eltrombopag versus standard first-line pharmacological management for newly diagnosed immune thrombocytopaenia (ITP) in children: study protocol. View Abstract
Response to rituximab in children and adults with immune thrombocytopenia (ITP). View Abstract
Definition of a critical bleed in patients with immune thrombocytopenia: Communication from the ISTH SSC Subcommittee on Platelet Immunology. View Abstract
The SSC platelet immunology register of VITT and VIITP: Toward standardization of laboratory and clinical parameters. View Abstract
Pyruvate kinase deficiency in children. View Abstract
Extensive variability in platelet, bleeding, and QOL outcome measures in adult and pediatric ITP: Communication from the ISTH SSC subcommittee on platelet immunology. View Abstract
Recommendations for the clinical and laboratory diagnosis of VITT against COVID-19: Communication from the ISTH SSC Subcommittee on Platelet Immunology. View Abstract
Preoperative hematocrit and platelet count are associated with blood loss during spinal fusion for children with neuromuscular scoliosis. View Abstract
Quality of life is an important indication for second-line treatment in children with immune thrombocytopenia. View Abstract
Refractory autoimmune cytopenias in pediatric Evans syndrome with underlying systemic immune dysregulation. View Abstract
Comorbidities and complications in adults with pyruvate kinase deficiency. View Abstract
International survey on Helicobacter pylori testing in patients with immune thrombocytopenia: Communication of the platelet immunology scientific and standardization committee. View Abstract
Tapering thrombopoietin receptor agonists in primary immune thrombocytopenia: Expert consensus based on the RAND/UCLA modified Delphi panel method. View Abstract
Management of pyruvate kinase deficiency in children and adults. View Abstract
The variable manifestations of disease in pyruvate kinase deficiency and their management. View Abstract
Immune dysregulation and multisystem inflammatory syndrome in children (MIS-C) in individuals with haploinsufficiency of SOCS1. View Abstract
Pyruvate kinase deficiency in a newborn with extramedullary hematopoiesis in the skin. View Abstract
Characterization of the severe phenotype of pyruvate kinase deficiency. View Abstract
Fatigue in children and adolescents with immune thrombocytopenia. View Abstract
COVID-19 presenting with autoimmune hemolytic anemia in the setting of underlying immune dysregulation. View Abstract
The pyruvate kinase (PK) to hexokinase enzyme activity ratio and erythrocyte PK protein level in the diagnosis and phenotype of PK deficiency. View Abstract
The role of romiplostim for pediatric patients with immune thrombocytopenia. View Abstract
The variable manifestations of disease in pyruvate kinase deficiency and their management. View Abstract
Genotype-phenotype correlation and molecular heterogeneity in pyruvate kinase deficiency. View Abstract
Development of the pyruvate kinase deficiency diary and pyruvate kinase deficiency impact assessment: Disease-specific assessments. View Abstract
Genetic variants in toll-like receptor 4 are associated with lack of steroid-responsiveness in pediatric ITP patients. View Abstract
American Society of Hematology 2019 guidelines for immune thrombocytopenia. View Abstract
Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency. View Abstract
Second-line treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes. View Abstract
Pediatric Hematology. View Abstract
Association of a positive direct antiglobulin test with chronic immune thrombocytopenia and use of second line therapies in children: A multi-institutional review. View Abstract
How we manage patients with pyruvate kinase deficiency. View Abstract
The burden of disease in pyruvate kinase deficiency: Patients' perception of the impact on health-related quality of life. View Abstract
Prevalence and management of iron overload in pyruvate kinase deficiency: report from the Pyruvate Kinase Deficiency Natural History Study. View Abstract
Ofatumumab for acute treatment and prophylaxis of a patient with multiple relapses of acquired thrombotic thrombocytopenic purpura. View Abstract
Red Blood Cell Enzyme Disorders. View Abstract
Physician decision making in selection of second-line treatments in immune thrombocytopenia in children. View Abstract
Clinical spectrum of pyruvate kinase deficiency: data from the Pyruvate Kinase Deficiency Natural History Study. View Abstract
The use of prophylactic anticoagulation during induction and consolidation chemotherapy in adults with acute lymphoblastic leukemia. View Abstract
Platelet Function in ITP, Independent of Platelet Count, Is Consistent Over Time and Is Associated with Both Current and Subsequent Bleeding Severity. View Abstract
Predictors of remission in children with newly diagnosed immune thrombocytopenia: Data from the Intercontinental Cooperative ITP Study Group Registry II participants. View Abstract
Second-line therapies in immune thrombocytopenia. View Abstract
Increasing observation rates in low-risk pediatric immune thrombocytopenia using a standardized clinical assessment and management plan (SCAMP® ). View Abstract
Exome sequencing results in successful diagnosis and treatment of a severe congenital anemia. View Abstract
Thrombopoietin Receptor Agonist Use in Children: Data From the Pediatric ITP Consortium of North America ICON2 Study. View Abstract
Vitamin B12 Deficiency Presenting with Neurological Dysfunction in an Adolescent. View Abstract
Erythrocyte pyruvate kinase deficiency: 2015 status report. View Abstract
Thrombopoietin-receptor agonists in children with immune thrombocytopenia. View Abstract
Platelet function tests, independent of platelet count, are associated with bleeding severity in ITP. View Abstract
Image-guided core needle biopsy in the diagnosis of malignant lymphoma. View Abstract
The utility of the DDAVP challenge test in children with low von Willebrand factor. View Abstract
Treatment and outcomes of immune cytopenias following solid organ transplant in children. View Abstract
The use of erythropoietin-stimulating agents versus supportive care in newborns with hereditary spherocytosis: a single centre's experience. View Abstract
Standardized clinical assessment and management plans (SCAMPs): perspectives on a new method to understand treatment decisions and outcomes in immune thrombocytopenia. View Abstract
A phase 2 study of the safety, tolerability, and pharmacodynamics of FBS0701, a novel oral iron chelator, in transfusional iron overload. View Abstract
Trends in anti-D immune globulin for childhood immune thrombocytopenia: usage, response rates, and adverse effects. View Abstract
Applicability of 2009 international consensus terminology and criteria for immune thrombocytopenia to a clinical pediatric population. View Abstract
Response to steroids predicts response to rituximab in pediatric chronic immune thrombocytopenia. View Abstract
The frequency and management of asparaginase-related thrombosis in paediatric and adult patients with acute lymphoblastic leukaemia treated on Dana-Farber Cancer Institute consortium protocols. View Abstract
Unsuspected pulmonary emboli in pediatric oncology patients: detection with MDCT. View Abstract
Genetic studies in pediatric ITP: outlook, feasibility, and requirements. View Abstract
Resolution of cerebral artery stenosis in a child with sickle cell anemia treated with hydroxyurea. View Abstract
Compliance with immunizations in splenectomized individuals with hereditary spherocytosis. View Abstract
Rituximab for adolescents with haemophilia and high titre inhibitors. View Abstract
Cardiac presentation of ALK positive anaplastic large cell lymphoma. View Abstract
Ivermectin is better than benzyl benzoate for childhood scabies in developing countries. View Abstract
Transmembrane versus soluble stem cell factor expression in human testis. View Abstract
Fludarabine, cytarabine, G-CSF and idarubicin (FLAG-IDA) for the treatment of poor-risk myelodysplastic syndromes and acute myeloid leukaemia. View Abstract
Ligand linked assembly of Scapharca dimeric hemoglobin. View Abstract