Sirolimus is effective for refractory/relapsed/intolerant acquired pure red cell aplasia: results of a prospective single-institutional trial

Huang, Yuzhou; Chen, Miao; Yang, Chen; Ruan, Jing; Wang, Shuqing; Han, Bing

doi:10.1038/s41375-022-01532-1

Cited by 12 publications

(2 citation statements)

References 33 publications

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“…Recently, a retrospective evaluation (n=64) from China reported successful treatment of PRCA with sirolimus-based therapy in terms of hemoglobin improvement. 11 Daratumumab demonstrated rapid and sustained response in treatment-refractory acquired idiopathic PRCA in a 74-year-old patient. 12 Daratumumab also successfully managed a patient of post-transplant PRCA with responses observed within 3 weeks.…”

Section: Discussionmentioning

confidence: 91%

Idiopathic pure red cell aplasia: A case series from India

Mahajan,

Goel,

Patel

et al. 2023

Asian J Med Sci

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Pure red cell aplasia (PRCA) is a rare hematologic disorder characterized by normocytic normochromic anemia with reticulocytopenia and depleted erythroid precursors. It is reported mainly from western countries. We report our experience in two patients of PRCA from India, the clinical characteristics, differential diagnosis, and their management. Our patients showed improvements following treatment with corticosteroids, blood transfusion, and/or rituximab.

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Section: Discussionmentioning

confidence: 91%

Idiopathic pure red cell aplasia: A case series from India

Mahajan,

Goel,

Patel

et al. 2023

Asian J Med Sci

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“…Sirolimus (rapamycin), a lipophilic macrolide antibiotic synthesized by streptomyces hygroscopicus , has strong immunosuppressive and anti-cell proliferation activities and has been used for antitumor therapy and post-transplantation immunosuppression [ 5 , 6 ]. We recently demonstrated that sirolimus is an effective treatment for patients with refractory /relapsed/intolerant aPRCA [ 7 ]. Even patients who are refractory to CsA may respond to sirolimus, indicating that sirolimus may have a different mechanism of action from that of CsA.…”

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confidence: 99%