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Safety profile of mycophenolate mofetil

Bone Marrow Transplantation volume 26, pages 1362–1363 (2000)Cite this article

In the May 2000 issue of Bone Marrow Transplantation, in ‘Mycophenolate mofetil (MMF) as therapy for refractory chronic GVHD (cGVHD) in children receiving bone marrow transplantation’ Busca et al1 have stated that six out of 14 patients treated with MMF experienced adverse events with predominating gastrointestinal symptoms. In addition MMF was discontinued after 35–180 days in six patients by parental choice (two patients), liver toxicity (one patient), poor compliance (two patients) and no response (one patient). However, hepatic dysfunction related to MMF in one patient persisted after MMF discontinuation and histological evaluation revealed characteristic bile duct abnormalities and portal inflammation. In the discussion, Busca et al stated: ‘to our knowledge, severe liver toxicity related to MMF has been reported in one study only5’. We would like to stress that the safety profile of mycophenolate mofetil in the setting of hematopoietic stem cell transplantation should be further evaluated. In our paper published in the May 1998 issue of Bone Marrow Transplantation2 we stated that no nephrotoxicity or liver toxicity were noted in our patients except for one, who had extensive cGVHD and developed progressive liver toxicity on MMF therapy. However, the progressive liver toxicity in this particular patient was most likely to have been due to progressive multiorgan failure rather than to MMF. Indeed, this patient died from progressive multiorgan failure related to extensive chronic GVHD and the treatment with MMF was an attempt to improve the patient's condition.

The paper by Busca et al raises the critical issue of differentiating the adverse effects of MMF from the clinical signs of severe GVHD; very few studies have addressed the issue of the effect of MMF in the treatment of GVHD (Table 1), however addressing primarily the efficacy of MMF. MMF is an immunosuppressant that selectively inhibits proliferation of T- and B-lymphocytes and is successfully used for the prevention of acute rejection in primary cadaveric renal allograft recipients.34 The study of the US Renal Transplant Mycophenolate Mofetil Study Group3 demonstrated that MMF administered at a dosage of 2 g is better tolerated compared with 3 g. The most frequently reported adverse events were anemia, hypertension and diarrhea. In addition, esophagitis, gastritis and gastrointestinal hemorrhages were observed. The tricontinental mycophenolate mofetil renal transplantation study group reported similar adverse effects,4 these being more severe in patients receiving MMF 3 g. No other specific organ toxicity was observed in either treatment group (MMF 2 g and MMF 3 g). The most common adverse event in pediatric renal transplant recipients was anemia, and only a small percentage of patients developed diarrhea and leukopenia.5 In addition MMF is used successfully for the treatment1267 and prophylaxis89 of acute GVHD after hematopoietic stem cell transplantation. The safety profile of MMF in hematopoietic stem cell transplantation revealed8 that the relative scores for renal, cardiac pulmonary and hepatic toxicity were comparable in the group of patients receiving MMF/CsA and those receiving MTX/CsA for the prophylaxis of GVHD.

Table 1  Reported studies of adverse effects following the treatment and prophylaxis of GVHD with MMF
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References

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  1. Clinic for Bone Marrow Transplantation and Haematology/Oncology, Dr Ottmar-Kohler Str 2, Idar-Oberstein, 55743, Germany

    N Basara & AA Fauser

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Basara, N., Fauser, A. Safety profile of mycophenolate mofetil. Bone Marrow Transplant 26, 1362–1363 (2000). https://doi.org/10.1038/sj.bmt.1702723

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