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Blood, 1 February 2006, Vol. 107, No. 3, pp. 1233-1236.
Prepublished online as a Blood First Edition Paper on October 11, 2005; DOI 10.1182/blood-2005-05-1819.
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Submitted May 5, 2005
Accepted September 18, 2005
Stem cell transplantation with reduced intensity conditioning for haemophagocytic lymphohistiocytosis
Nichola Cooper, Kanchan Rao, Kimberly Gilmour, Lema Hadad, Stuart Adams, Cathy Cale, Graham Davies, David Webb, Paul Veys, and Persis J Amrolia*
Bone marrow transplant, haematology and molecular immunology, Great Ormond Street Hospital, London, United Kingdom
* Corresponding author; email: amrolp1{at}gosh.nhs.uk.
Allogeneic stem cell transplant (SCT) is curative for haemophagocytic lymphohistiocytosis (HLH). However, patients frequently have significant pre-transplant morbidity and there is high transplant related mortality (TRM). Because primary HLH is caused by immune dysregulation, a reduced intensity conditioned (RIC) regimen might be sufficient for cure, while decreasing the TRM. 12 patients with HLH underwent RIC SCT from a matched family/unrelated or haploidentical donor. 11 were conditioned with fludarabine/melphalan with additional busulphan for haplo-identical grafts. 1 received fludarabine and 2GyTBI. All patients engrafted at a median of 14 days. 9 of 12(75%) are alive and in CR a median of 30 (range 9 to 73) months post SCT. 3 died from pneumonitis (n=2) and hepatic rupture (n=1). 4 developed acute GVHD and 3 have chronic GVHD. 3 of 9 survivors have mixed chimerism but remain disease-free. In summary, RIC compares favorably to conventional SCT with long-term disease control in surviving patients despite a significant incidence of mixed chimerism.

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