Paroxysmal Nocturnal Hemoglobinuria Clinical Trial
Official title:
Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory Classic Paroxysmal Nocturnal Hemoglobinuria ,a Prospective Study
Classical paroxysmal nocturnal hemoglobinuria(PNH) is mainly characterized by hemolysis and thrombosis, which reduced patients ' quality of life(QoL) greatly and even lead to death. Glucocorticoids and symptomatic supportive therapy are traditional treatments and the response rate is far from satisfactory. Eculizumab is an effective therapy but it is expensive and not available in China mainland.The investigators aim to explore the efficacy and safety of sirolimus for refractory classic PNH.
Classical paroxysmal nocturnal hemoglobinuria(PNH) is mainly characterized by hemolysis and
thrombosis, which reduced patients ' quality of life(QoL) greatly and even lead to death.
There is no ideal therapy except for eculizumab, expensive and not available in China
mainland. Glucocorticoids and symptomatic supportive therapy are traditional treatments. The
response rate is 30%, far from satisfactory. In recent years, T lymphocyte-mediated
destruction of normal hematopoietic stem cells have been reported to involve the pathogenesis
of PNH, making immunomodulatory drugs be potential effective treatments.
Sirolimus (rapamycin), produced by the bacterium Streptomyces hygroscopicus, is a mammalian
target of Rapamycin (mTOR) inhibitor. mTOR is a serine/threonine kinase that regulates cell
growth, proliferation, metabolism and survival. It has two interacting complex, mTORC1 and
mTORC2. Sirolimus primarily inhibits mTORC1, has been demonstrated for its immunomodulatory
effects and ability to improve hematopoietic stem cell function. Recently, sirolimus has been
reported to be effective and well tolerated in the treatment of immune-mediated cytopenias,
even in multi-immunosuppressants resistant patients. In addition, classical PNH patients have
a higher risk of thrombosis especially in refractory ones and it is reasonable to use
low-dose warfarin in the management of patients with classic refractory PNH.
In this study, it is anticipated to evaluate the effect of sirolimus combined with low-dose
warfarin on patients with refractory classic PNH. The adverse effects and QoL on different
time points were documented.
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