Rachael Grace

Publications

1. Working toward international consensus defining pediatric "refractory ITP". Blood Adv. 2025 Mar 11; 9(5):1244-1245. View Abstract
2. Correlation of Genetic Mutation With Outcomes in Children With Hereditary Spherocytosis Undergoing Partial Splenectomy: A Multicentre Study. J Pediatr Surg. 2025 Apr; 60(4):162229. View Abstract
3. T cell and autoantibody profiling for primary immune regulatory disorders. medRxiv. 2025 Jan 27. View Abstract
4. Treatment of Critical Bleeds in Patients With Immune Thrombocytopenia: A Systematic Review. Eur J Haematol. 2025 Mar; 114(3):458-468. View Abstract
5. Genetic variants in canonical Wnt signaling pathway associated with pediatric immune thrombocytopenia. Blood Adv. 2024 11 12; 8(21):5529-5538. View Abstract
6. What is in a name: defining pediatric refractory ITP. Blood Adv. 2024 10 08; 8(19):5112-5117. View Abstract
7. Quality of life in childhood immune thrombocytopenia: Revision of the Kids' ITP Tools (KIT). Br J Haematol. 2024 Oct; 205(4):1489-1496. View Abstract
8. The 2022 review of the 2019 American Society of Hematology guidelines on immune thrombocytopenia. Blood Adv. 2024 07 09; 8(13):3578-3582. View Abstract
9. Comorbidities and complications in adult and paediatric patients with pyruvate kinase deficiency: Analysis from the Peak Registry. Br J Haematol. 2024 Aug; 205(2):613-623. View Abstract
10. Mitapivat improves ineffective erythropoiesis and iron overload in adult patients with pyruvate kinase deficiency. Blood Adv. 2024 05 28; 8(10):2433-2441. View Abstract
11. Clinically meaningful improvements in patient-reported outcomes in mitapivat-treated patients with pyruvate kinase deficiency. Am J Hematol. 2024 07; 99(7):1415-1419. View Abstract
12. Bone mineral density in adult patients with pyruvate kinase deficiency on long-term mitapivat treatment. Haematologica. 2024 03 01; 109(3):963-967. View Abstract
13. Diagnosis and management of pyruvate kinase deficiency: international expert guidelines. Lancet Haematol. 2024 Mar; 11(3):e228-e239. View Abstract
14. Pyruvate kinase activators for treatment of pyruvate kinase deficiency. Hematology Am Soc Hematol Educ Program. 2023 Dec 08; 2023(1):97-106. View Abstract
15. Psychometric validation of the Pyruvate Kinase Deficiency Diary and Pyruvate Kinase Deficiency Impact Assessment in adults in the phase 3 ACTIVATE trial. J Patient Rep Outcomes. 2023 Nov 09; 7(1):112. View Abstract
16. HLA antibodies in fetal and neonatal alloimmune thrombocytopenia. Transfusion. 2023 06; 63(6):1141-1149. View Abstract
17. The Pyruvate Kinase Deficiency Global Longitudinal (Peak) Registry: rationale and study design. BMJ Open. 2023 03 23; 13(3):e063605. View Abstract
18. Updates and advances in pyruvate kinase deficiency. Trends Mol Med. 2023 05; 29(5):406-418. View Abstract
19. Early-onset reduced bone mineral density in patients with pyruvate kinase deficiency. Am J Hematol. 2023 03; 98(3):E57-E60. View Abstract
20. Pediatric refractory immune thrombocytopenia: A systematic review. Pediatr Blood Cancer. 2023 03; 70(3):e30173. View Abstract
21. Long-term risk of developing immune thrombocytopenia and hematologic neoplasia in adults with mild thrombocytopenia. Blood. 2022 12 29; 140(26):2849-2852. View Abstract
22. Utilization of an ITP quality improvement pathway improves adherence to management guidelines. Pediatr Blood Cancer. 2023 03; 70(3):e30074. View Abstract
23. An update on pediatric ITP: differentiating primary ITP, IPD, and PID. Blood. 2022 08 11; 140(6):542-555. View Abstract
24. SARS-CoV-2 vaccination in pediatric patients with immune thrombocytopenia. Pediatr Blood Cancer. 2022 07; 69(7):e29760. View Abstract
25. Diagnosis, monitoring, and management of pyruvate kinase deficiency in children. Pediatr Blood Cancer. 2022 08; 69(8):e29696. View Abstract
26. Mitapivat versus Placebo for Pyruvate Kinase Deficiency. N Engl J Med. 2022 04 14; 386(15):1432-1442. View Abstract
27. Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency. Blood Adv. 2022 03 22; 6(6):1844-1853. View Abstract
28. Thrombopoietin receptor agonists and rituximab for treatment of pediatric immune thrombocytopenia: A systematic review and meta-analysis of prospective clinical trials. Pediatr Blood Cancer. 2022 03; 69(3):e29447. View Abstract
29. Characteristics and outcomes of autoimmune hemolytic anemia after pediatric allogeneic stem cell transplant. Pediatr Blood Cancer. 2022 01; 69(1):e29410. View Abstract
30. Phase 3 randomised trial of eltrombopag versus standard first-line pharmacological management for newly diagnosed immune thrombocytopaenia (ITP) in children: study protocol. BMJ Open. 2021 08 27; 11(8):e044885. View Abstract
31. Response to rituximab in children and adults with immune thrombocytopenia (ITP). Res Pract Thromb Haemost. 2021 Aug; 5(6):e12587. View Abstract
32. Definition of a critical bleed in patients with immune thrombocytopenia: Communication from the ISTH SSC Subcommittee on Platelet Immunology. J Thromb Haemost. 2021 08; 19(8):2082-2088. View Abstract
33. The SSC platelet immunology register of VITT and VIITP: Toward standardization of laboratory and clinical parameters. J Thromb Haemost. 2021 08; 19(8):2094-2095. View Abstract
34. Pyruvate kinase deficiency in children. Pediatr Blood Cancer. 2021 09; 68(9):e29148. View Abstract
35. Extensive variability in platelet, bleeding, and QOL outcome measures in adult and pediatric ITP: Communication from the ISTH SSC subcommittee on platelet immunology. J Thromb Haemost. 2021 09; 19(9):2348-2354. View Abstract
36. Recommendations for the clinical and laboratory diagnosis of VITT against COVID-19: Communication from the ISTH SSC Subcommittee on Platelet Immunology. J Thromb Haemost. 2021 06; 19(6):1585-1588. View Abstract
37. Preoperative hematocrit and platelet count are associated with blood loss during spinal fusion for children with neuromuscular scoliosis. J Perioper Pract. 2022 04; 32(4):74-82. View Abstract
38. Quality of life is an important indication for second-line treatment in children with immune thrombocytopenia. Pediatr Blood Cancer. 2021 06; 68(6):e29023. View Abstract
39. Refractory autoimmune cytopenias in pediatric Evans syndrome with underlying systemic immune dysregulation. Eur J Haematol. 2021 Jun; 106(6):783-787. View Abstract
40. Comorbidities and complications in adults with pyruvate kinase deficiency. Eur J Haematol. 2021 Apr; 106(4):484-492. View Abstract
41. International survey on Helicobacter pylori testing in patients with immune thrombocytopenia: Communication of the platelet immunology scientific and standardization committee. J Thromb Haemost. 2021 01; 19(1):287-296. View Abstract
42. Tapering thrombopoietin receptor agonists in primary immune thrombocytopenia: Expert consensus based on the RAND/UCLA modified Delphi panel method. Res Pract Thromb Haemost. 2021 Jan; 5(1):69-80. View Abstract
43. Management of pyruvate kinase deficiency in children and adults. Blood. 2020 09 10; 136(11):1241-1249. View Abstract
44. The variable manifestations of disease in pyruvate kinase deficiency and their management. Haematologica. 2020 09 01; 105(9):2229-2239. View Abstract
45. Immune dysregulation and multisystem inflammatory syndrome in children (MIS-C) in individuals with haploinsufficiency of SOCS1. J Allergy Clin Immunol. 2020 11; 146(5):1194-1200.e1. View Abstract
46. Pyruvate kinase deficiency in a newborn with extramedullary hematopoiesis in the skin. Blood. 2020 08 06; 136(6):770. View Abstract
47. Characterization of the severe phenotype of pyruvate kinase deficiency. Am J Hematol. 2020 Oct; 95(10):E281-E285. View Abstract
48. Fatigue in children and adolescents with immune thrombocytopenia. Br J Haematol. 2020 10; 191(1):98-106. View Abstract
49. COVID-19 presenting with autoimmune hemolytic anemia in the setting of underlying immune dysregulation. Pediatr Blood Cancer. 2020 09; 67(9):e28382. View Abstract
50. The pyruvate kinase (PK) to hexokinase enzyme activity ratio and erythrocyte PK protein level in the diagnosis and phenotype of PK deficiency. Br J Haematol. 2021 03; 192(6):1092-1096. View Abstract
51. The role of romiplostim for pediatric patients with immune thrombocytopenia. Ther Adv Hematol. 2020; 11:2040620720912992. View Abstract
52. The variable manifestations of disease in pyruvate kinase deficiency and their management. Haematologica. 2020 Mar 12. View Abstract
53. Genotype-phenotype correlation and molecular heterogeneity in pyruvate kinase deficiency. Am J Hematol. 2020 05; 95(5):472-482. View Abstract
54. Development of the pyruvate kinase deficiency diary and pyruvate kinase deficiency impact assessment: Disease-specific assessments. Eur J Haematol. 2020 May; 104(5):427-434. View Abstract
55. Genetic variants in toll-like receptor 4 are associated with lack of steroid-responsiveness in pediatric ITP patients. Am J Hematol. 2020 04; 95(4):395-400. View Abstract
56. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019 12 10; 3(23):3829-3866. View Abstract
57. Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency. N Engl J Med. 2019 09 05; 381(10):933-944. View Abstract
58. Second-line treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes. Am J Hematol. 2019 07; 94(7):741-750. View Abstract
59. Pediatric Hematology. Hematol Oncol Clin North Am. 2019 06; 33(3):xiii-xiv. View Abstract
60. Association of a positive direct antiglobulin test with chronic immune thrombocytopenia and use of second line therapies in children: A multi-institutional review. Am J Hematol. 2019 04; 94(4):461-466. View Abstract
61. How we manage patients with pyruvate kinase deficiency. Br J Haematol. 2019 03; 184(5):721-734. View Abstract
62. The burden of disease in pyruvate kinase deficiency: Patients' perception of the impact on health-related quality of life. Eur J Haematol. 2018 Dec; 101(6):758-765. View Abstract
63. Prevalence and management of iron overload in pyruvate kinase deficiency: report from the Pyruvate Kinase Deficiency Natural History Study. Haematologica. 2019 02; 104(2):e51-e53. View Abstract
64. Ofatumumab for acute treatment and prophylaxis of a patient with multiple relapses of acquired thrombotic thrombocytopenic purpura. J Thromb Thrombolysis. 2018 Jul; 46(1):81-83. View Abstract
65. Red Blood Cell Enzyme Disorders. Pediatr Clin North Am. 2018 06; 65(3):579-595. View Abstract
66. Physician decision making in selection of second-line treatments in immune thrombocytopenia in children. Am J Hematol. 2018 07; 93(7):882-888. View Abstract
67. Clinical spectrum of pyruvate kinase deficiency: data from the Pyruvate Kinase Deficiency Natural History Study. Blood. 2018 05 17; 131(20):2183-2192. View Abstract
68. The use of prophylactic anticoagulation during induction and consolidation chemotherapy in adults with acute lymphoblastic leukemia. J Thromb Thrombolysis. 2018 Feb; 45(2):306-314. View Abstract
69. Platelet Function in ITP, Independent of Platelet Count, Is Consistent Over Time and Is Associated with Both Current and Subsequent Bleeding Severity. Thromb Haemost. 2018 01; 118(1):143-151. View Abstract
70. Predictors of remission in children with newly diagnosed immune thrombocytopenia: Data from the Intercontinental Cooperative ITP Study Group Registry II participants. Pediatr Blood Cancer. 2018 Jan; 65(1). View Abstract
71. Second-line therapies in immune thrombocytopenia. Hematology Am Soc Hematol Educ Program. 2016 Dec 02; 2016(1):698-706. View Abstract
72. Increasing observation rates in low-risk pediatric immune thrombocytopenia using a standardized clinical assessment and management plan (SCAMP® ). Pediatr Blood Cancer. 2017 05; 64(5). View Abstract
73. Exome sequencing results in successful diagnosis and treatment of a severe congenital anemia. Cold Spring Harb Mol Case Stud. 2016 Jul; 2(4):a000885. View Abstract
74. Thrombopoietin Receptor Agonist Use in Children: Data From the Pediatric ITP Consortium of North America ICON2 Study. Pediatr Blood Cancer. 2016 08; 63(8):1407-13. View Abstract
75. Vitamin B12 Deficiency Presenting with Neurological Dysfunction in an Adolescent. Pediatr Neurol. 2016 09; 62:66-70. View Abstract
76. Erythrocyte pyruvate kinase deficiency: 2015 status report. Am J Hematol. 2015 Sep; 90(9):825-30. View Abstract
77. Thrombopoietin-receptor agonists in children with immune thrombocytopenia. Lancet. 2015 Oct 24; 386(10004):1606-9. View Abstract
78. Platelet function tests, independent of platelet count, are associated with bleeding severity in ITP. Blood. 2015 Aug 13; 126(7):873-9. View Abstract
79. Image-guided core needle biopsy in the diagnosis of malignant lymphoma. Eur J Surg Oncol. 2015 Jul; 41(7):852-8. View Abstract
80. The utility of the DDAVP challenge test in children with low von Willebrand factor. Br J Haematol. 2015 Sep; 170(6):884-6. View Abstract
81. Treatment and outcomes of immune cytopenias following solid organ transplant in children. Pediatr Blood Cancer. 2015 02; 62(2):214-218. View Abstract
82. The use of erythropoietin-stimulating agents versus supportive care in newborns with hereditary spherocytosis: a single centre's experience. Eur J Haematol. 2014 Aug; 93(2):161-4. View Abstract
83. Standardized clinical assessment and management plans (SCAMPs): perspectives on a new method to understand treatment decisions and outcomes in immune thrombocytopenia. Semin Hematol. 2013 Jan; 50 Suppl 1:S31-8. View Abstract
84. A phase 2 study of the safety, tolerability, and pharmacodynamics of FBS0701, a novel oral iron chelator, in transfusional iron overload. Blood. 2012 Apr 05; 119(14):3263-8. View Abstract
85. Trends in anti-D immune globulin for childhood immune thrombocytopenia: usage, response rates, and adverse effects. Am J Hematol. 2012 Mar; 87(3):315-7. View Abstract
86. Applicability of 2009 international consensus terminology and criteria for immune thrombocytopenia to a clinical pediatric population. Pediatr Blood Cancer. 2012 Feb; 58(2):216-20. View Abstract
87. Response to steroids predicts response to rituximab in pediatric chronic immune thrombocytopenia. Pediatr Blood Cancer. 2012 Feb; 58(2):221-5. View Abstract
88. 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. Br J Haematol. 2011 Feb; 152(4):452-9. View Abstract
89. Unsuspected pulmonary emboli in pediatric oncology patients: detection with MDCT. AJR Am J Roentgenol. 2010 May; 194(5):1216-22. View Abstract
90. Genetic studies in pediatric ITP: outlook, feasibility, and requirements. Ann Hematol. 2010 Jul; 89 Suppl 1:S95-103. View Abstract
91. Resolution of cerebral artery stenosis in a child with sickle cell anemia treated with hydroxyurea. Am J Hematol. 2010 Feb; 85(2):135-7. View Abstract
92. Compliance with immunizations in splenectomized individuals with hereditary spherocytosis. Pediatr Blood Cancer. 2009 Jul; 52(7):865-7. View Abstract
93. Rituximab for adolescents with haemophilia and high titre inhibitors. Haemophilia. 2006 May; 12(3):218-22. View Abstract
94. Cardiac presentation of ALK positive anaplastic large cell lymphoma. Eur J Haematol. 2005 Dec; 75(6):511-4. View Abstract
95. Ivermectin is better than benzyl benzoate for childhood scabies in developing countries. J Paediatr Child Health. 2002 Aug; 38(4):401-4. View Abstract
96. Transmembrane versus soluble stem cell factor expression in human testis. J Androl. 2000 Jul-Aug; 21(4):579-85. View Abstract
97. Fludarabine, cytarabine, G-CSF and idarubicin (FLAG-IDA) for the treatment of poor-risk myelodysplastic syndromes and acute myeloid leukaemia. Br J Haematol. 1997 Dec; 99(4):939-44. View Abstract
98. Ligand linked assembly of Scapharca dimeric hemoglobin. J Biol Chem. 1997 Feb 28; 272(9):5689-94. View Abstract
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