Treatment of Aplastic Anemia with Supportive Care, Hgfs, and Immunosuppressives

Doney, Kristine C.

Abstract

This project continues to develop effective therapies for patients with aplastic anemia who are not candidates for genotypically HLA-identical donor bone marrow transplants. Previous studies performed in Seattle have shown that immunosuppressive regimens including anti-human thymocyte globulin (ATG), high or low-dose corticosteroids and androgens, achieves long-term survival in 44% of patients. In an effort to improve these results the role of hematopoietic growth factors given singly or in combination along with immunosuppressive therapy will be evaluated. Long-term follow-up of patients will determine the incidence of late complications such as recurrent aplasia, myelodysplastic syndrome, and paroxysmal nocturnal hemoglobinuria (PNH). Statistical analyses will be used to (1) identify clinical or laboratory parameters associated with response to therapy and (2) compare the results of immunosuppressive therapy to those of marrow transplantation.

Funding Agency

• Agency: National Institute of Health (NIH)
• Institute: National Heart, Lung, and Blood Institute (NHLBI)
• Type: Research Program Projects (P01)
• Project #: 5P01HL036444-12
• Application #: 3844478
• Support Year: 12
• Fiscal Year: 1992

Institution

• Name: Fred Hutchinson Cancer Research Center
• DUNS #: 075524595
• City: Seattle
• State: WA
• Country: United States
• Zip Code: 98109

Publications

McCune, Jeannine S; Storer, Barry; Thomas, Sushma et al. (2018) Inosine Monophosphate Dehydrogenase Pharmacogenetics in Hematopoietic Cell Transplantation Patients. Biol Blood Marrow Transplant 24:1802-1807

Thakar, M S; Bonfim, C; Walters, M C et al. (2017) Dose-adapted post-transplant cyclophosphamide for HLA-haploidentical transplantation in Fanconi anemia. Bone Marrow Transplant 52:570-573

Burroughs, Lauri M; Shimamura, Akiko; Talano, Julie-An et al. (2017) Allogeneic Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for Treatment of Marrow Failure Disorders. Biol Blood Marrow Transplant 23:1669-1677

Hoffmeister, P A; Storer, B E; Syrjala, K L et al. (2016) Physician-diagnosed depression and suicides in pediatric hematopoietic cell transplant survivors with up to 40 years of follow-up. Bone Marrow Transplant 51:153-6

Gallo, S; Woolfrey, A E; Burroughs, L M et al. (2016) Marrow grafts from HLA-identical siblings for severe aplastic anemia: does limiting the number of transplanted marrow cells reduce the risk of chronic GvHD? Bone Marrow Transplant 51:1573-1578

Festuccia, Moreno; Deeg, H Joachim; Gooley, Theodore A et al. (2016) Minimal Identifiable Disease and the Role of Conditioning Intensity in Hematopoietic Cell Transplantation for Myelodysplastic Syndrome and Acute Myelogenous Leukemia Evolving from Myelodysplastic Syndrome. Biol Blood Marrow Transplant 22:1227-1233

Vaughn, J E; Anwer, F; Deeg, H J (2016) Treatment of refractory ITP and Evans syndrome by haematopoietic cell transplantation: is it indicated, and for whom? Vox Sang 110:5-11

Aki, S Z; Inamoto, Y; Carpenter, P A et al. (2016) Confounding factors affecting the National Institutes of Health (NIH) chronic Graft-Versus-Host Disease Organ-Specific Score and global severity. Bone Marrow Transplant 51:1350-1353

Khera, Nandita; Gooley, Ted; Flowers, Mary E D et al. (2016) Association of Distance from Transplantation Center and Place of Residence on Outcomes after Allogeneic Hematopoietic Cell Transplantation. Biol Blood Marrow Transplant 22:1319-1323

Karoopongse, Ekapun; Marcondes, A Mario; Yeung, Cecilia et al. (2016) Disruption of Iron Regulation after Radiation and Donor Cell Infusion. Biol Blood Marrow Transplant 22:1173-1181