Rachael Grace

Publications

1. T cell and autoantibody profiling for primary immune regulatory disorders. medRxiv. 2025 Jan 27. View Abstract
2. International Consensus in Aiming to Define "Refractory ITP". Blood Adv. 2025 Jan 09. View Abstract
3. Treatment of Critical Bleeds in Patients With Immune Thrombocytopenia: A Systematic Review. Eur J Haematol. 2024 Nov 18. View Abstract
4. Genetic variants in canonical Wnt signaling pathway associated with pediatric immune thrombocytopenia. Blood Adv. 2024 11 12; 8(21):5529-5538. View Abstract
5. What is in a name: defining pediatric refractory ITP. Blood Adv. 2024 10 08; 8(19):5112-5117. View Abstract
6. 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
7. 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
8. 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
9. 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
10. 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
11. 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
12. Diagnosis and management of pyruvate kinase deficiency: international expert guidelines. Lancet Haematol. 2024 Mar; 11(3):e228-e239. View Abstract
13. Pyruvate kinase activators for treatment of pyruvate kinase deficiency. Hematology Am Soc Hematol Educ Program. 2023 Dec 08; 2023(1):97-106. View Abstract
14. 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
15. HLA antibodies in fetal and neonatal alloimmune thrombocytopenia. Transfusion. 2023 06; 63(6):1141-1149. View Abstract
16. The Pyruvate Kinase Deficiency Global Longitudinal (Peak) Registry: rationale and study design. BMJ Open. 2023 03 23; 13(3):e063605. View Abstract
17. Updates and advances in pyruvate kinase deficiency. Trends Mol Med. 2023 05; 29(5):406-418. View Abstract
18. Early-onset reduced bone mineral density in patients with pyruvate kinase deficiency. Am J Hematol. 2023 03; 98(3):E57-E60. View Abstract
19. Pediatric refractory immune thrombocytopenia: A systematic review. Pediatr Blood Cancer. 2023 03; 70(3):e30173. View Abstract
20. 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
21. Utilization of an ITP quality improvement pathway improves adherence to management guidelines. Pediatr Blood Cancer. 2023 03; 70(3):e30074. View Abstract
22. An update on pediatric ITP: differentiating primary ITP, IPD, and PID. Blood. 2022 08 11; 140(6):542-555. View Abstract
23. SARS-CoV-2 vaccination in pediatric patients with immune thrombocytopenia. Pediatr Blood Cancer. 2022 07; 69(7):e29760. View Abstract
24. Diagnosis, monitoring, and management of pyruvate kinase deficiency in children. Pediatr Blood Cancer. 2022 08; 69(8):e29696. View Abstract
25. Mitapivat versus Placebo for Pyruvate Kinase Deficiency. N Engl J Med. 2022 04 14; 386(15):1432-1442. View Abstract
26. 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
27. 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
28. Characteristics and outcomes of autoimmune hemolytic anemia after pediatric allogeneic stem cell transplant. Pediatr Blood Cancer. 2022 01; 69(1):e29410. View Abstract
29. 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
30. Response to rituximab in children and adults with immune thrombocytopenia (ITP). Res Pract Thromb Haemost. 2021 Aug; 5(6):e12587. View Abstract
31. 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
32. 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
33. Pyruvate kinase deficiency in children. Pediatr Blood Cancer. 2021 09; 68(9):e29148. View Abstract
34. 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
35. 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
36. 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
37. 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
38. Refractory autoimmune cytopenias in pediatric Evans syndrome with underlying systemic immune dysregulation. Eur J Haematol. 2021 Jun; 106(6):783-787. View Abstract
39. Comorbidities and complications in adults with pyruvate kinase deficiency. Eur J Haematol. 2021 Apr; 106(4):484-492. View Abstract
40. 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
41. 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
42. Management of pyruvate kinase deficiency in children and adults. Blood. 2020 09 10; 136(11):1241-1249. View Abstract
43. The variable manifestations of disease in pyruvate kinase deficiency and their management. Haematologica. 2020 09 01; 105(9):2229-2239. View Abstract
44. 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
45. Pyruvate kinase deficiency in a newborn with extramedullary hematopoiesis in the skin. Blood. 2020 08 06; 136(6):770. View Abstract
46. Characterization of the severe phenotype of pyruvate kinase deficiency. Am J Hematol. 2020 Oct; 95(10):E281-E285. View Abstract
47. Fatigue in children and adolescents with immune thrombocytopenia. Br J Haematol. 2020 10; 191(1):98-106. View Abstract
48. COVID-19 presenting with autoimmune hemolytic anemia in the setting of underlying immune dysregulation. Pediatr Blood Cancer. 2020 09; 67(9):e28382. View Abstract
49. 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
50. The role of romiplostim for pediatric patients with immune thrombocytopenia. Ther Adv Hematol. 2020; 11:2040620720912992. View Abstract
51. The variable manifestations of disease in pyruvate kinase deficiency and their management. Haematologica. 2020 Mar 12. View Abstract
52. Genotype-phenotype correlation and molecular heterogeneity in pyruvate kinase deficiency. Am J Hematol. 2020 05; 95(5):472-482. View Abstract
53. 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
54. 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
55. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019 12 10; 3(23):3829-3866. View Abstract
56. Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency. N Engl J Med. 2019 09 05; 381(10):933-944. View Abstract
57. 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
58. Pediatric Hematology. Hematol Oncol Clin North Am. 2019 06; 33(3):xiii-xiv. View Abstract
59. 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
60. How we manage patients with pyruvate kinase deficiency. Br J Haematol. 2019 03; 184(5):721-734. View Abstract
61. 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
62. 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
63. 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
64. Red Blood Cell Enzyme Disorders. Pediatr Clin North Am. 2018 06; 65(3):579-595. View Abstract
65. 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
66. 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
67. 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
68. 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
69. 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
70. Second-line therapies in immune thrombocytopenia. Hematology Am Soc Hematol Educ Program. 2016 Dec 02; 2016(1):698-706. View Abstract
71. 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
72. 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
73. 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
74. Vitamin B12 Deficiency Presenting with Neurological Dysfunction in an Adolescent. Pediatr Neurol. 2016 09; 62:66-70. View Abstract
75. Erythrocyte pyruvate kinase deficiency: 2015 status report. Am J Hematol. 2015 Sep; 90(9):825-30. View Abstract
76. Thrombopoietin-receptor agonists in children with immune thrombocytopenia. Lancet. 2015 Oct 24; 386(10004):1606-9. View Abstract
77. Platelet function tests, independent of platelet count, are associated with bleeding severity in ITP. Blood. 2015 Aug 13; 126(7):873-9. View Abstract
78. Image-guided core needle biopsy in the diagnosis of malignant lymphoma. Eur J Surg Oncol. 2015 Jul; 41(7):852-8. View Abstract
79. 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
80. Treatment and outcomes of immune cytopenias following solid organ transplant in children. Pediatr Blood Cancer. 2015 02; 62(2):214-218. View Abstract
81. 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
82. 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
83. 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
84. 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
85. 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
86. Response to steroids predicts response to rituximab in pediatric chronic immune thrombocytopenia. Pediatr Blood Cancer. 2012 Feb; 58(2):221-5. View Abstract
87. 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
88. Unsuspected pulmonary emboli in pediatric oncology patients: detection with MDCT. AJR Am J Roentgenol. 2010 May; 194(5):1216-22. View Abstract
89. Genetic studies in pediatric ITP: outlook, feasibility, and requirements. Ann Hematol. 2010 Jul; 89 Suppl 1:S95-103. View Abstract
90. 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
91. Compliance with immunizations in splenectomized individuals with hereditary spherocytosis. Pediatr Blood Cancer. 2009 Jul; 52(7):865-7. View Abstract
92. Rituximab for adolescents with haemophilia and high titre inhibitors. Haemophilia. 2006 May; 12(3):218-22. View Abstract
93. Cardiac presentation of ALK positive anaplastic large cell lymphoma. Eur J Haematol. 2005 Dec; 75(6):511-4. View Abstract
94. Ivermectin is better than benzyl benzoate for childhood scabies in developing countries. J Paediatr Child Health. 2002 Aug; 38(4):401-4. View Abstract
95. Transmembrane versus soluble stem cell factor expression in human testis. J Androl. 2000 Jul-Aug; 21(4):579-85. View Abstract
96. 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
97. Ligand linked assembly of Scapharca dimeric hemoglobin. J Biol Chem. 1997 Feb 28; 272(9):5689-94. View Abstract
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