A clinical trial evaluating the efficacy of everolimus in controlling systemic mastocytosis (SM) has been recently updated with a complete list of adverse events.

The nonrandomized, open-label study enrolled 10 participants who received 10 mg of oral everolimus daily for 30 days. The primary outcome of the study was the number of participants with objective response (ie, change in serum tryptase level or bone marrow mast cell percentage), which was evaluated monthly for the first 3 months and every 3 months afterward.

Two participants reported serious adverse events, including nausea, vomiting, and diarrhea. Moreover, all participants reported nonserious adverse events such as anemia (10%), thrombocytopenia (10%), neutropenia (30%), hyperglycemia (10%), hyperlipidemia (30%), diarrhea (40%), mucositis (70%), nausea (30%), vomiting (10%), fatigue (50%), fever (10%), headache (40%), and cough (20%). No participant discontinued the study.

In addition, secondary outcome measures were safety of the treatment in patients with SM, time to progression and duration of response following treatment, and the change in pertinent biological markers during the therapy.

Read more about SM treatment

The study included those with mast cell leukemia, aged 18 years or older, with Eastern Cooperative Oncology Group performance status of 2 or less, adequate liver function, and normal prothrombin time/partial thromboplastin time/international normalized ratio. Furthermore, participants had to have a minimum of a 2-week interval since any major surgery or completion of radiation.

Patients treated with any conventional or investigational therapy for SM within the preceding 4 weeks, as well as those under chronic treatment with systemic steroids or another immunosuppressive agent, were excluded from the study. Also excluded were pregnant or breastfeeding women, patients with certain malignancies within the past 3 years or other concurrent severe and/or uncontrolled medical disease that could compromise participation, patients with a known history of human immunodeficiency virus seropositivity, impaired gastrointestinal function, or a diagnosed gastrointestinal disease that may significantly alter drug absorption, patients with a bleeding diathesis or on oral antivitamin K medication, patients who received prior treatment with an mTOR inhibitor or with a known hypersensitivity to everolimus, other rapamycins, or its excipients, and patients unwilling to or unable to comply with the protocol.

Results were first published in April 2011. The trial was initiated in April 2007 and concluded in October 2009. The trial was sponsored by the MD Anderson Cancer Center, in Houston, Texas, and counted with the collaboration of Novartis Pharmaceuticals.


Everolimus (RAD001) as therapy for patients with systemic mastocytosis. ClinicalTrials.gov. March 21, 2007. Updated October 25, 2022. Accessed October 26, 2022.