Open-label, Single-arm, Phase II, Prospective, Pilot Study of Tocilizumab in Patients With Erdheim-Chester Disease
Inclusion Criteria:
- patients >= 18 years of age able to understand and sign an informed consent;
- histologically proven diagnosis of Erdheim-Chester disease ;
- an advanced disease not limited to the skeleton, with at least one measurable lesion;
- if females of childbearing potential, a negative pregnancy test and willingness to
adhere to a highly effective contraceptive method of birth control for the duration
of the study;
Exclusion Criteria:
- history of hypersensitivity to tocilizumab or to any of the excipients;
- severe infections requiring hospitalization or antibiotic therapy in the 30 days
before the enrollment in the study;
- active tuberculosis, listeriosis, histoplasmosis, sepsis, abscesses, opportunistic
infections; active hepatitis B or C virus infection;
- past history of tuberculosis (as documented by a positive purified protein derivative
(PPD) skin test and/or a positive Quantiferon test and/or a chest X- ray), in the
absence of a documented and appropriate administration of a specific treatment for
latent tuberculosis;
- history of human immunodeficiency virus (HIV) infection;
- past history (< 5 years before enrollment) of a lymphoproliferative disorder or of a
solid cancer (excluding cured basal cell or squamous cell carcinoma of the skin);
- moderate or severe heart failure (NYHA class III/IV), uncontrolled diabetes mellitus
or other diseases that -according to the physician in charge of the protocol- may be
of harm to the patient, if he/she would enroll in the study;
- history of alcohol and/or drug abuse;
- prior treatment with alkylating drugs (chlorambucil, cyclophosphamide);
- serum creatinine > 1.6 mg/dL in female patients or > 1.9 mg/dL in male patients,
aspartate aminotransferase and/or alanine aminotransferase > 3 x upper limit of
normal ; platelet < 100.000/fL; hemoglobin < 8.5 g/dL; white blood cell count <
1000/fL; lymphocyte < 500/fL; total bilirubin > 2.0 mg/dL