Bone Marrow Failure Medication
- Author: Srikanth Nagalla, MBBS, MS, FACP; Chief Editor: Koyamangalath Krishnan, MD, FRCP, FACP more...
The approach to bone marrow failure depends on which mechanism is thought to predominate in the patient. If an immune mechanism is suspected, an immunosuppressive agent is used. Hematopoietic growth factors and androgens also have been tried in an effort to stimulate hematopoiesis.
As previously mentioned, androgens were used in the past for treatment of bone marrow failure, but most are masculinizing and poorly tolerated by females and children. Danazol is a nonmasculinizing androgen that may be useful. The response rate is limited to approximately 45%, and results may require 6-10 months of therapy.
These are used to manipulate the bone marrow microenvironment and eliminate any immune-mediated bone marrow suppression. Intensive immunosuppression using a combination of ALG and cyclosporine has resulted in hematologic remission rates of 70-80% in patients with aplastic anemia.
This agent, an antibody to T cells, is used as an immunosuppressive agent. Because it is extracted from horse serum, serum sickness may be induced when the drug is administered.
Cyclosporine A is a cyclic polypeptide that suppresses some humoral immunity and, to a greater extent, cell-mediated immune reactions, such as delayed hypersensitivity, allograft rejection, experimental allergic encephalomyelitis, and graft versus host disease. For children and adults, the dosing should be based on ideal body weight.
Methylprednisolone decreases inflammation by suppressing the migration of polymorphonuclear leukocytes and reversing increased capillary permeability.
Prednisone is used as an immunosuppressant in the treatment of autoimmune disorders. By reversing increased capillary permeability and suppressing polymorphonuclear leukocyte activity, it may decrease inflammation.
These agents push the resting hematopoietic stem cells into cycle, making them more responsive to differentiation by hematopoietic growth factors. They also stimulate endogenous secretion of erythropoietin
Danocrine is an attenuated androgen that does not have adverse virilizing and masculinizing effects. It increases levels of the C4 component of the complement.
Young NS. Acquired bone marrow failure. Handin RI, Stossel TP, Lux SE, eds. Blood: Principles and Practice of Hematology. Philadelphia, Pa: JB Lippincott; 1995. 293-365.
Richardson C, Yan S, Vestal CG. Oxidative stress, bone marrow failure, and genome instability in hematopoietic stem cells. Int J Mol Sci. 2015 Jan 22. 16 (2):2366-85. [Medline].
Chung NG, Kim M. Current insights into inherited bone marrow failure syndromes. Korean J Pediatr. 2014 Aug. 57 (8):337-44. [Medline].
Townsley DM, Dumitriu B, Young NS. Bone marrow failure and the telomeropathies. Blood. 2014 Oct 30. 124 (18):2775-83. [Medline].
Alter BP. Bone marrow failure: a child is not just a small adult (but an adult can have a childhood disease). Hematology Am Soc Hematol Educ Program. 2005. 96-103. [Medline].
Townsley DM, Desmond R, Dunbar CE, Young NS. Pathophysiology and management of thrombocytopenia in bone marrow failure: possible clinical applications of TPO receptor agonists in aplastic anemia and myelodysplastic syndromes. Int J Hematol. 2013 Jul. 98(1):48-55. [Medline].
Grewal SS, Kahn JP, MacMillan ML, Ramsay NK, Wagner JE. Successful hematopoietic stem cell transplantation for Fanconi anemia from an unaffected HLA-genotype-identical sibling selected using preimplantation genetic diagnosis. Blood. 2004 Feb 1. 103(3):1147-51. [Medline].
Molldrem JJ, Leifer E, Bahceci E, Saunthararajah Y, Rivera M, Dunbar C, et al. Antithymocyte globulin for treatment of the bone marrow failure associated with myelodysplastic syndromes. Ann Intern Med. 2002 Aug 6. 137(3):156-63. [Medline].
Parikh S, Bessler M. Recent insights into inherited bone marrow failure syndromes. Curr Opin Pediatr. 2012 Feb. 24(1):23-32. [Medline].