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Rituximab therapy for autoimmune blood disorders

Autoimmune diseases

Autoimmune hematological diseases are conditions where the body’s immune system produces antibodies that attack and damage specific proteins and blood cells in the bloodstream.

Although these are rare conditions, the more common of these include the following disorders:

  • Autoimmune hemolytic anemia – anti-red blood cells antibodies attach to the surface of the red blood cells and damage them to the point where they rupture causing the contents of the cell to spill into the bloodstream. These patients experience signs and symptoms of anemia because the hemoglobin proteins that are supposed to carry oxygen to the tissues and organs of the body are no longer functional. These patients will also have a yellow discoloration of their skin and/or eyes due to bilirubin entering the bloodstream from the ruptured cells, and the urine will be darkly colored as a result of the circulating hemoglobin filtered out into this bodily fluid.1
  • Primary immune thrombocytopenia – antibodies are produced where they attack the platelet cells in the bloodstream. The function of the platelets is to help prevent prolonged and excessive bleeding by clumping up and clotting blood vessel injuries. Patients with this condition may experience signs and symptoms such as spontaneous formation of large bruises (purpura) or smaller bruises (petechiae) on the limbs, uncontrolled and/or excessive bleeding from the gums, lips or nostrils, or in severe cases spontaneous formation of blood masses (hematomas) can occur in the mouth or other mucous membranes of the body.2
  • Thrombotic thrombocytopenic purpura – this condition affects the same platelet cells as in primary immune thrombocytopenia but the effect is the opposite. Here, antibodies cause microscopic clots to form in the small blood vessels throughout the body which can cause obstruction of oxygen and nutrient transport to essential tissues causing organ damage.3
  • Acquired hemophilia – a rare form of hemophilia since this condition is associated with a hereditary link as it is mainly passed on to children of parents with the defective gene that has the potential to cause the disorder to develop in their offspring. Here, antibodies have formed that attack and damage the clotting factors responsible for making sure that blood clots properly. The result is that affected patients may experience problems such as persistent bleeding that’s difficult to control, and an increased risk of bleeding into joints or even the brain.4

The current treatment therapies for these conditions include suppressing the immune system from producing antibodies with the administration of steroid medications, and if that doesn’t work then antibody-damaging (cytotoxic) drugs. If these medications are ineffective, then healthcare professionals suggest surgically removing the spleen (splenectomy).5

One medication, in particular, may be an effective form of treatment in patients who develop the mentioned autoimmune hematological conditions.


Rituximab is an anti-CD20 monoclonal antibody which has the specific action of reducing the presence of B-cells in the bloodstream which are involved in the production of antibodies that damage blood cells and proteins. This results in a decreased amount of problematic antibodies and for a new population of healthy B-cells to be produced from the lymphoid stem cells.6

In a study performed on the effect of rituximab on autoimmune hematological diseases, it was demonstrated that the medication had a positive effect on the outcomes of patients irrespective of their age and how many times they were treated with more conventional therapies before the rituximab was initiated. The researchers suggested that the medication is an effective and well-tolerated treatment in managing signs and symptoms of autoimmune hematological diseases.7

Side effects of rituximab may occur within a couple of hours after administration of the drug and can include issues such as itchiness, skin rashes, shortness of breath, or low blood pressure.8

Rituximab was approved for medicinal use in many other conditions including the mentioned autoimmune hematological diseases in 1997 and it is on the World Health Organization’s List of Essential Medicines which includes the safest and most effective medications needed in healthcare systems worldwide.9

It is also a very cost-effective medication which makes it an ideal treatment for rare autoimmune conditions, especially those that don’t respond to conventional therapies or are severe in nature.


  1. Sawitsky A, Ozaeta PB (June 1970). "Disease-associated autoimmune hemolytic anemia"Bull N Y Acad Med46(6): 411–26. PMC1749710 . PMID 5267234.
  2. Rodeghiero, F; Stasi, R; Gernsheimer, T; Michel, M; Provan, D; Arnold, DM; Bussel, JB; Cines, DB; Chong, BH; Cooper, N; Godeau, B; Lechner, K; Mazzucconi, MG; McMillan, R; Sanz, MA; Imbach, P; Blanchette, V; Kühne, T; Ruggeri, M; George, JN (12 March 2009). "Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group". Blood. 113(11): 2386–93.
  3. "Thrombotic thrombocytopenic purpura". Dorland's Medical Dictionary for Health Consumers. Philadelphia, PA: Saunders. 2007.
  4. "What Causes Hemophilia?"NHLBI. July 13, 2013. Archivedfrom the original on 8 September 2016.
  5. Go, R. S., Winters, J. L., & Kay, N. E. (2017). How I treat autoimmune hemolytic anemia. Blood, (), blood-2016-11-693689. Accessed April 28, 2017
  6. Bosch, Xavier; Ramos-Casals, Manuel; Khamashta, Munther A. (2013). Drugs Targeting B-Cells in Autoimmune Diseases. Springer Science & Business Media. pp. 1–4.
  7. Barcellini, W., Zanella, A. (2011). Rituximab therapy for autoimmune haematological disease. European Journal of Internal Medicine. 22: 220–229.
  8. "Rituximab". The American Society of Health-System Pharmacists. Archivedfrom the original on 27 March 2016. Retrieved 8 December2016.
  9. "WHO Model List of Essential Medicines (19th List)"(PDF). World Health Organization. April 2015.