Lenalidomide has been licenced for the treatment of
multiple myeloma and, in 2012, it is used as a standard treatment of relapses of the disease. Literature contains a number of publications on the effects of
lenalidomide in
myelodysplastic syndrome, in
malignant lymphomas and chronic B lymphocytic leukaemia. The effects of the
drug in
rare diseases, however, have not been investigated so far. In this paper, we summarize our experience with
lenalidomide in rare blood disorders. We observed an excellent effect of
lenalidomide in multifocal aggressive, repeatedly relapsing
Langerhans cell histiocytosis where it led to complete remission. This patient was treated with
2-chlorodeoxyadenosine and with CHOEP (
cyclophosphamide,
etoposide,
doxorubicin,
vincristine and
prednisone)
chemotherapy and high dose BEAM
chemotherapy with
autologous transplantation of haematopoietic tissue for an early disease relapse. Following another early relapse, the patient was treated with
lenalidomide (25 mg). Treatment with
lenalidomide induced complete remission on PET-CT. The patient was consolidated during the remission with a reduced intensity conditioning regimen and
allogeneic transplantation of haematopoietic tissue. Following
allogeneic transplantation, the patient has been
in full remission for 10 months. We further showed an excellent effect of
lenalidomide in multicentric
Castleman disease with generalized involvement of lymphatic nodes, B symptoms and
vasculitis. The patient was first treated
R-CHOP chemotherapy (
rituximab,
cyclophosphamide,
adriamycin,
vincristine and
prednisone). Due to a lack of efficacy, this was changed to the CVD combination (
cyclophosphamide,
thalidomide, dexamethazone). This treatment delivered complete remission but was complicated by
thalidomide-associated neuropathy. Due to persistent neuropathy,
thalidomide could not be used to manage further relapse and thus
lenalidomide (25 mg, 11 cycles) was used. The patient has been in complete PET-CT remission for 7 months following this treatment. We observed partial efficacy in
Erdheim-Chester disease. We used
2-chlorodeoxyadenosine as part of initial treatment that delivered partial regression of brain infiltrates only; fluorodeoxyglucose accumulation in the bones has not changed.
Lenalidomide 25 mg was used as second line treatment. This led to complete regression of CNS infiltrates on MRI but fluorodeoxyglucose accumulation in bone lesions did not change. Regression of clinical signs and regression of
fibrosis of retroperitoneum was achieved with an ongoing treatment with
anakinra. A patient with multiple
angiomatosis affecting the abdominal cavity, mediastinum and vertebrae and digestive tract had been stabilized with
zoledronate (4 mg once every 2 months) and
thalidomide (100 - 200 mg/den) for several years. However, several years of this treatment led to severe neuropathy. Consequently, we attempted to substitute
thalidomide for
lenalidomide. However, 10 mg of
lenalidomide alone was not sufficiently effective and thus low dose of 50 mg of
thalidomide was added. Combined treatment with
zoledronate,
lenalidomide 10 mg/day and
thalidomide 50 mg/day stabilized the condition for 9 months. Due to relapsed gastrointestinal
bleeding the treatment had to be changed after 9 months to
thalidomide 100 mg/day and
Sandostatin 0.1 mg twice daily s.c. A patient with osteosclerotic myeloma and
POEMS syndrome was initially treated with CAD
chemotherapy (
cyclophosphamide, adriamycine and dexamethazone) that was followed by tandem high dose
chemotherapy (
melphalan 100 mg/m2) and
autologous transplantation. Treatment with
thalidomide was given due to insufficient efficacy but was not tolerated.
Lenalidomide was administered as the fourth line treatment. Even though literature describes remission of
POEMS syndrome following
lenalidomide, four cycles did not lead to remission in our patient.
CONCLUSION: