Article

Use of mycophenolate mofetil for chronic, refractory immune cytopenias in children with autoimmune lymphoproliferative syndrome

Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases/NIH, 10 Center Drive, Bethesda, MD 20892, USA.
British Journal of Haematology (Impact Factor: 4.96). 06/2005; 129(4):534-8. DOI: 10.1111/j.1365-2141.2005.05496.x
Source: PubMed

ABSTRACT Autoimmune lymphoproliferative syndrome (ALPS) is a disorder of apoptosis associated most often with heritable FAS mutations leading to lymphadenopathy, hypersplenism and chronic refractory autoimmune cytopenias. Mycophenolate mofetil (MMF) was used to treat cytopenias in 13 ALPS patients aged 9 months to 17 years from a cohort of 118 children (aged < 18 years) and 82 adults. Twelve responded for a median follow-up of 49 weeks (range 38-240 weeks), defined by maintenance of adequate blood counts and reduction in dosage or cessation of other immunosuppressive agents. This preliminary experience suggests that MMF may spare steroid usage in patients with ALPS-associated cytopenias.

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Available from: V Koneti Rao, Aug 28, 2015
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    • "Infection occurred in 6/19 patients after a median period of 70 weeks (Gomez-Almaguer et al., 2010). Other reports have documented an initial response rate of 78–92% for refractory autoimmune cytopenias treated with mycophenolate mofetil with no significant adverse events reported (Kotb et al., 2005; Rao et al., 2005). Thus, the approach to treating autoimmune cytopenias in CVID is not dissimilar to the treatment of immune competent patients (Wang and Cunningham-Rundles, 2005). "
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    • "For accurate diagnosis of ALPS, these tests should be performed by laboratories familiar with the test methods and in which local normal values are established [88]. The best frontline treatment of patients who have ALPS is with mycophenolate mofetil (MMF); in the largest series of ALPS reported to date of treatment with this immunosuppressive agent, a response rate of 92% was observed [89]. Splenectomy should be avoided in ALPS cases because of the high risk for overwhelming postsplenectomy sepsis. "
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    • "Moreover, the effects of rituximab are generally relatively transient; patients that do respond will probably relapse and with the potential risk of CVID, we recommend avoiding use of rituximab in ALPS patients. MMF was found to be effective in another series (Koneti Rao et al, 2005). While we have had less success overall with this drug, we have also seen good responses in a few children with ALPS treated with MMF (unpublished data). "
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