POEMS Syndrome Treatment & Management

Updated: Jun 13, 2018
  • Author: Joanna L Chan, MD; Chief Editor: Dirk M Elston, MD  more...
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Treatment

Medical Care

The treatment of POEMS syndrome depends on the treatment of the underlying plasma cell disorder. Most patients are treated with a combination of medical, surgical, and adjuvant therapies. [28]

The current mainstays of treatment for patients with diffuse disease include combinations of corticosteroids, low-dose alkylators, and peripheral blood stem cell transplantation following high-dose chemotherapy. [29, 30] Some caution should be used in selecting the chemotherapeutic regimen to avoid worsening of the polyneuropathy.

Widespread osteosclerotic lesions may benefit from systemic therapy, with approximately half the patients benefiting from melphalan and prednisone. Although about a quarter of patients respond to corticosteroids, relapses are common without additional treatment of the underlying plasma cell disorder. Intravenous immunoglobulin and plasmapheresis have not shown therapeutic benefit.

In a study from China, 31 patients received 12 cycles of oral melphalan (10 mg/m2 body surface area [BSA]) plus oral dexamethasone (40 mg/d) on days 1-4 of every 28 days. [31] Prophylaxis with a proton-pump inhibitor was added days -1 to 5. Doses were adjusted according to side effects and renal function. Good hematologic and neurologic responses were reported.

Some patients may consider high-dose chemotherapy followed by autologous hematopoietic stem cell transplantation. Preliminary studies report a mortality rate of 7.4% associated with the procedure, in addition to significant peritransplantation morbidity that often requires intubation, but nearly all survivors experienced benefit. A case report described the successful treatment of a patient with multicentric Castleman disease and POEMS syndrome with autologous hematopoietic stem cell transplantation, with subsequent improved nerve conduction and normalization of serum protein electrophoresis results. [32]

Treatment with high-dose chemotherapy followed by autologous peripheral blood stem cell transplantation may result in clinical improvements of polyneuropathy. [33]

Relapse after successful auto peripheral blood stem cell transplantation has been reported. [34]

A 2014 multicenter retrospective study in Japan has shown positive results for autologous stem cell transplantation (ACST) in the treatment of patients with POEMS syndrome in terms of long-term survival and quality of life. [35]

In a series of 30 POEMS syndrome patients treated at the Mayo Clinic in Rochester, Minn, engraftment syndrome was reported in approximately half the patients, with symptoms including fever (93%), diarrhea (77%), weight gain (53%), and rash (43%), in addition to a 3% treatment-related mortality rate. Baseline splenomegaly was predictive of a complicated peritransplantation course. [36]

Kojima et al [37] describe clinical remission of more than 20 months for one patient who received high-dose chemotherapy with autologous CD34+ -purged stem cells.

The clinical course of one patient improved after administration of thalidomide, which has well-described antiangiogenetic, antiproliferative, and anticytokine characteristics. [38]

A case report of one patient with POEMS syndrome and elevated VEGF levels describes successful treatment with bevacizumab, an antiangiogenesis monoclonal antibody directed against VEGF. This treatment was associated with reduced edema and less painful neuropathy. [39] Another patient treated with bevacizumab (10 mg/kg) twice monthly had decreased pain after one month, with improvements in breathing and walking within 10 weeks. These subjective changes were associated with a drop in VEGF levels and clinical improvements measured by pulmonary function tests, electromyographic studies, and nerve conduction studies. In this patient, neither hepatomegaly nor skin abnormalities were modified. [40]

Responses to bevacizumab reportedly have been variable. In one patient treated 7 years after diagnosis, no clinical improvement was reported despite lowered VEGF levels, which the authors thought may be due to aberrant angiogenesis that had already developed systemically. [41] Another POEMS patient treated with 2 courses of bevacizumab (5 mg/kg) had decreased VEGF levels but worsening paresis, and this patient developed severe capillary leak syndrome with edema, ascites, and pleural effusion. These authors encourage caution prior to starting bevacizumab therapy because a sudden decrease in VEGF levels may result in apoptosis of endothelial cells, possibly increasing capillary leakiness. [42] This approach is supported by another report of a patient treated by bevacizumab who developed diarrhea, generalized edema, pulmonary hypertension, and systemic inflammation and who subsequently died from multiorgan failure.

One patient improved after receiving 9 cycles of lenalidomide given daily for 21 days of a 28-day cycle with once-weekly dexamethasone. The dosage of lenalidomide was gradually increased from 15 mg to the standard 25-mg dose. Lenalidomide is cytotoxic to malignant plasma cells in addition to being immune modulatory. [43] In 2009, a case report also described lenalidomide therapy in a patient with kappa restriction. [44]  A 2015 prospective phase II trial of lenalidomide combined with dexamethasone (LEN-DEX) reported a promising reduction of VEGF to normal levels and recovery of neurological function in many patients, with minimal adverse effects. [45]

Sanada et al [46] report the clinical remission and correspondingly decreased VEGF serum concentrations of one patient with POEMS syndrome and renal lesions after treatment with high-dose melphalan therapy followed by autologous blood stem cell transplantation.

A single patient with POEMS syndrome associated with 2 monoclonal gammopathies (IgG-kappa and IgA-gamma) has been reported, in whom high doses of all-trans -retinoic acid was potentially beneficial. [47]

Two case reports describe successful therapy with bortezomib. [48, 49]

For patients with POEMS and pulmonary hypertension, high-dose steroid treatment appears to provide improvement as confirmed by sequential hemodynamic studies. [50, 51]

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Surgical Care

Surgical excision of isolated plasmacytomas may result in complete resolution of the syndrome.

Radiation therapy of solitary osteosclerotic lesions is first-line therapy for patients with an isolated plasmacytoma.

Radiation therapy in dosages of 40-50 cGy to limited areas can improve osteosclerotic lesions in more than half the patients. More than 6 months may elapse before clinically apparent improvement is observed, with benefit observed even 2-3 years after therapy. Other authors recommend systemic therapy for patients whose disease process fails to stabilize 3-6 months after completing radiation therapy.

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Consultations

Care of patients with POEMS syndrome is coordinated through many specialists because of the extent of possible symptoms associated with the disorder.

Once the diagnosis is clinically suggested, the patient should be evaluated by a neurologist, a hematologist, a dermatologist, and an endocrinologist to define the extent of disease.

Referral to a pulmonologist or a nephrologist should be guided by the patient's symptomatology.

A surgeon or a radiation oncologist may be needed for coordination of treatment.

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Long-Term Monitoring

Even if the plasmacytoma completely responds to treatment, patients need long-term follow-up care because some symptoms, such as neurologic defects and functional loss due to tightening of the skin, may be permanent.

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