Paroxysmal Nocturnal Hemoglobinuria Medication

Updated: Jun 29, 2016
  • Author: Emmanuel C Besa, MD; Chief Editor: Koyamangalath Krishnan, MD, FRCP, FACP  more...
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Medication

Medication Summary

The drugs used in treatment of paroxysmal nocturnal hemoglobinuria (PNH) include the following:

  • Eculizumab, to stop hemolysis
  • Recombinant erythropoietin or androgens, to stimulate erythropoiesis
  • Anticoagulants, to treat thrombotic complications
  • Immunosuppressive agents, to stimulate hematopoiesis in the aplastic phase
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Androgens

Class Summary

Androgens are used to stimulate erythropoiesis by increasing endogenous levels of erythropoietin and by enhancing the response of precursor cells to the growth factor.

Attenuated androgens, such as danazol, are recommended for use in women, as the attenuated androgen has fewer adverse virilizing and masculinizing effects.

Oxymetholone (Anadrol-50)

Anabolic and androgenic derivative of testosterone in an oral formulation.

Used to stimulate erythropoiesis by increasing endogenous levels of erythropoietin and by enhancing the response of precursor cells to the growth factor.

Stanozolol (Winstrol)

Anabolic and androgenic derivative of testosterone in an oral formulation.

Danazol (Danocrine)

Synthetic steroid analogue, derived from ethisterone, with strong antigonadotropic activity (inhibits LH and FSH) and weak androgenic action without adverse virilizing and masculinizing effects. Increases levels of C4 component of the complement. May push the resting hematopoietic stem cells into cycle, making them more responsive to differentiation by hematopoietic growth factors. May also stimulate endogenous secretion of erythropoietin.

May impair clearance of immunoglobulin-coated platelets and decreases autoantibody production.

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Immunosuppressive Agents

Class Summary

Antithymocyte globulin is an antiserum to human T cells prepared from horses or rabbits. [39] The mechanism of action of polyclonal antilymphocyte preparations to suppress immune responses is not fully understood.

Antithymocyte globulin; rabbit (Atgam)

Purified preparation of pasteurized polyclonal IgG obtained from rabbits immunized against human thymocytes (T cells) for IV use. This preparation has replaced the Upjohn preparation Atgam (horse serum) and is considered an equivalent.

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Complement Inhibitor

Class Summary

The C-5 inhibitor eculizumab has been designated as an orphan drug for the treatment paroxysmal nocturnal hemoglobinuria (PNH).

Eculizumab (Soliris)

Orphan drug indicated for treatment of paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis. Blocks complement-mediated destruction of PNH red blood cells. Inhibits C-5 component of complement system, thereby preventing final stages of complement activation.

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