Autoimmune cytopenia

Section editors
	Shruti Chaturvedi, MBBS, MSCI Johns Hopkins University Baltimore, MD LinkedIn		Benjamin Tillman, MD Vanderbilt University Nashville, TN

2 regimens on this page 2 variants on this page

This is an umbrella category that includes Felty's syndrome (autoimmune neutropenia), autoimmune thrombocytopenia (ITP), autoimmune hemolytic anemia (AIHA), and Evan's syndrome (AIHA & ITP). Please see the respective page(s) for disease-specific therapies. This page includes regimens that were used more broadly.

Relapsed or refractory

Alemtuzumab monotherapy

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Regimen

Study	Evidence	Efficacy
Willis et al. 2001	Phase II	ORR: 71%

Immunosuppressive therapy

• Alemtuzumab (Campath) as follows:
  • Test dose: 1 mg IV over 60 minutes once
  • Days 1 to 10: 10 mg IV over 4 hours once per day

10-day course

References

1. Willis F, Marsh JC, Bevan DH, Killick SB, Lucas G, Griffiths R, Ouwehand W, Hale G, Waldmann H, Gordon-Smith EC. The effect of treatment with Campath-1H in patients with autoimmune cytopenias. Br J Haematol. 2001 Sep;114(4):891-8. link to original article contains verified protocol PubMed

Sirolimus monotherapy

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Regimen

Study	Evidence
Bride et al. 2015	Phase II

Immunosuppressive therapy

• Sirolimus (Rapamune) 2 to 2.5 mg/m²/day PO
  • See manuscript for recommended dose adjustments

6-month course, extended for those with a favorable response

References

1. Bride KL, Vincent T, Smith-Whitley K, Lambert MP, Bleesing JJ, Seif AE, Manno CS, Casper J, Grupp SA, Teachey DT. Sirolimus is effective in relapsed/refractory autoimmune cytopenias: results of a prospective multi-institutional trial. Blood. 2016 Jan 7;127(1):17-28. Epub 2015 Oct 26. link to original article link to PMC article contains protocol PubMed