Experience with a novel efalizumab-based immunosuppressive regimen to facilitate single donor islet cell transplantation.

Experience with a novel efalizumab-based immunosuppressive regimen to facilitate single donor islet cell transplantation.
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DOI:
10.1111/j.1600-6143.2010.03212.x
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发表时间:
2010-09
期刊:
American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons
影响因子:
--
通讯作者:
Larsen CP
Larsen CP
中科院分区:
其他
文献类型:
--
作者:
Turgeon NA;Avila JG;Cano JA;Hutchinson JJ;Badell IR;Page AJ;Adams AB;Sears MH;Bowen PH;Kirk AD;Pearson TC;Larsen CP

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Islet transplantation is an experimental therapy for selected patients with type 1-diabetes (T1DM). It remains limited by immunosuppressive drug toxicity, progressive loss of insulin independence, allosensitization, and the need for multiple islet donors. We describe our experience with an efalizumab-based immunosuppressive regimen as compared to the prevailing standard regimen, the Edmonton protocol. Twelve patients with T1DM received islet transplants: 8 were treated with the Edmonton protocol; 4 were treated with daclizumab induction, a 6-month course of tacrolimus, and maintenance with efalizumab and mycophenolate mofetil. The primary endpoint was insulin independence after one islet infusion. Only 2 Edmonton protocol treated patients achieved the primary endpoint; 6 required islets from multiple donors, and all experienced leukopenia, mouth ulcers, anemia, diarrhea, and hypertransaminasemia. Four became allosensitized. All patients treated with the efalizumab-based regimen achieved insulin independence with normal hemoglobin A1c after a single islet cell infusion and remained insulin independent while on efalizumab. These patients experienced significantly fewer side effects and none became allosensitized. Trial continuation was terminated by withdrawal of efalizumab from the market. These data suggest that this efalizumab-based regimen prevents islet rejection, is well tolerated, and allows for single donor islet transplantation.
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