Autoimmune cytopenia

Section editor
	Benjamin Tillman, MD Vanderbilt University Nashville, TN, USA LinkedIn

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.

• We have moved How I Treat articles to a dedicated page.

Last updated on 2024-07-23: 2 regimens on this page 2 variants on this page

Relapsed or refractory

Alemtuzumab monotherapy

Regimen

Study	Dates of enrollment	Evidence	Efficacy
Willis et al. 2001	1995-11 to 2000-08	Phase 2	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 dosing details in manuscript have been reviewed by our editors PubMed

Sirolimus monotherapy

Regimen

Study	Dates of enrollment	Evidence	Efficacy
Bride et al. 2015	Not reported	Phase 2	Durable CR observed in patients with ALPS

Immunosuppressive therapy

• Sirolimus (Rapamune) 2 to 2.5 mg/m²/day PO

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

Dose and schedule modifications

• See manuscript for recommended dose adjustments

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 dosing details in abstract have been reviewed by our editors PubMed NCT00392951