Management of POEMS Syndrome
First-Line Treatment Based on Disease Extent
For localized disease with solitary or limited sclerotic bone lesions, radiation therapy is the definitive first-line treatment, achieving 97% 4-year overall survival and 52% 4-year failure-free survival. 1 This approach results in improvement in 50-70% of patients and should be used when there is no bone marrow involvement. 1, 2
For disseminated disease with diffuse sclerotic lesions or bone marrow involvement, systemic chemotherapy followed by autologous stem cell transplantation (ASCT) in eligible patients is the recommended approach. 1, 2
Systemic Therapy Regimens
Preferred First-Line Options
Melphalan-dexamethasone achieves 81% hematologic response and 100% improvement in neuropathy in prospective trials, making it a preferred regimen for disseminated disease. 1, 2
Lenalidomide-dexamethasone is preferred in patients with pre-existing neuropathy due to lower neurotoxicity risk compared to other agents. 2, 3
Bortezomib monotherapy achieved complete remission/very good partial remission in 69% of patients in real-world analysis, though it carries neuropathy risk. 4
Role of Autologous Stem Cell Transplantation
ASCT should be offered to eligible patients after induction chemotherapy, achieving 100% clinical improvement in transplant-eligible patients. 1, 2
Patients who proceeded to planned ASCT as part of front-line treatment demonstrated statistically superior progression-free survival and overall survival compared to non-ASCT treated patients (P=0.003). 4
Agents to Avoid
Thalidomide-based regimens should be avoided due to high neurotoxicity risk that can worsen pre-existing neuropathy. 1
Bortezomib-based combinations are not recommended as first-line due to induced neuropathy risk, despite high response rates. 1, 2, 5
Critical Treatment Principles
Timeline of Response
Neurologic improvement significantly lags behind hematologic response, with maximum neurologic response expected after 2-3 years of successful therapy. 1, 2, 3
Continue treatment even if immediate neurologic improvement is not apparent—do not prematurely discontinue therapy based on lack of early neurologic response. 2
Optimal response on FDG-PET may lag by 6-12 months after treatment. 1
Monitoring Parameters
Serum VEGF levels should be monitored as a marker of disease activity and treatment response. 1, 2, 6
The main goals of therapy are to attain complete hematologic and VEGF responses and to reduce symptoms. 3
Supportive Care Management
Endocrinopathy management should address thyroid, adrenal, and gonadal dysfunction. 1
Thrombotic risk management should monitor for thrombocytosis and consider prophylaxis. 1
Pulmonary and cardiac assessment should include regular monitoring with pulmonary function tests and echocardiography. 1
Diagnostic Distinction from Multiple Myeloma
Patients who otherwise satisfy criteria for myeloma but are symptomatic due to POEMS syndrome should be managed differently, taking into consideration the manifestations of the associated condition. 7 POEMS syndrome should be kept in the differential diagnosis when considering myeloma, as treatment approaches differ fundamentally. 7
Risk Factors for Poor Outcomes
Anemia, thrombocytopenia, and age over 60 were associated with negative impact on patient outcomes in multivariate analysis. 4
Low serum albumin, pleural effusion, pulmonary hypertension, and reduced estimated glomerular filtration rate are additional risk factors. 6, 5
Critical Pitfall to Avoid
Do not use radiation alone for disseminated disease—it is not curative and systemic therapy is mandatory. 2 Patients with diffuse sclerotic lesions or disseminated bone marrow involvement require systemic therapy, not localized radiation. 5