title: Salvage lenalidomide in four rare oncological diseases.
abstract: In rare disorders, there are often no standard therapy recommendations. Patients with refractory disease may require novel experimental approaches. Applied as second- up to fourth-line treatment, lenalidomide (10-25 mg perorally on days 1-21 in a 28-day cycle) was used in our cohort of four adult patients with aggressive, multisystem and relapsing diseases. Complete and long-lasting remissions (more than 1 year, no maintenance therapy) were achieved in patients with Langerhans cell histiocytosis (11 cycles, combination with dexamethasone and etoposide, consolidated by allogeneic blood stem cell transplant) and plasma-cell Castleman disease (15 cycles, monotherapy). Mixed response with complete disappearance of brain infiltrates was reached in Erdheim-Chester disease (6 cycles, monotherapy) and gastrointestinal bleeding was well controlled in multiple angiomatosis (9 cycles, combination with thalidomide). For disease activity evaluation each patient underwent fluorine-18-fluorodeoxyglucose positron emission tomography/computed tomography scan imaging, which was complemented by clinical and laboratory investigations.
authors.forename: Petr
authors.initials: P
authors.lastname: Szturz
authors.forename: Zdenek
authors.initials: Z
authors.lastname: Adam
authors.forename: Zdenek
authors.initials: Z
authors.lastname: Rehak
authors.forename: Renata
authors.initials: R
authors.lastname: Koukalova
authors.forename: Leos
authors.initials: L
authors.lastname: Kren
authors.forename: Mojmír
authors.initials: M
authors.lastname: Moulis
authors.forename: Marta
authors.initials: M
authors.lastname: Krejcí
authors.forename: Jiri
authors.initials: J
authors.lastname: Mayer
mesh_terms: D000328:Adult; D020533:Angiogenesis Inhibitors / Q000627:therapeutic use*; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D005871:Castleman Disease / Q000188:drug therapy* / Q000473:pathology; D057176:Compassionate Use Trials; D018572:Disease-Free Survival; D031249:Erdheim-Chester Disease / Q000188:drug therapy; D018380:Hematopoietic Stem Cell Transplantation; D006646:Histiocytosis, Langerhans-Cell / Q000188:drug therapy* / Q000473:pathology; D006801:Humans; D000077269:Lenalidomide; D008279:Magnetic Resonance Imaging; D008297:Male; D008875:Middle Aged; D049268:Positron-Emission Tomography; D035583:Rare Diseases / Q000188:drug therapy; D012189:Retrospective Studies; D016879:Salvage Therapy / Q000379:methods*; D013792:Thalidomide / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use*; D014057:Tomography, X-Ray Computed; D014182:Transplantation, Autologous; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D020533:Angiogenesis Inhibitors; D013792:Thalidomide; D000077269:Lenalidomide