Long-term follow-up on a patient with incomplete POEMS syndrome undergoing high-dose therapy and autologous blood stem cell transplantation

Jaccard et al recently reported a series of 5 patients with polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome who underwent high-dose chemotherapy followed by autologous stem cell transplantation.1 In 2001, first reports of single patients after autologous transplantations were published.2 3 So far, all published cases show dramatic improvement in clinical manifestations, especially of polyneuropathy, and no relapses have been reported after autologous stem cell transplantation. However, data on long-term follow-up are extremely rare. Median follow-up of patients published by Jaccard et al was 36 months, with the first patient being assessed 58 months after transplantation.

Here, we report a patient who developed multifocal osteosclerotic myeloma in 1994 at the age of 30. Osteosclerosis was diagnosed in the skull, both femura, and the pelvis, and the diagnosis was confirmed by biopsy. Bone marrow histology revealed no increase in the number of plasma cells. Due to progressive polyneuropathy predominating the lower limbs, the patient rapidly became paraplegic. Muscular strength of the arms was considerably reduced as well. All muscular stretch reflexes were absent. There was hypesthesia and pallhypesthesia of the lower extremities. Electroneurographic studies revealed that nerves of the lower extremities had no motorial or sensoric nerve action potentials, and F waves were absent. Also, nerves of the upper extremities showed electroneurographic abnormalities with reduction of nerve conduction velocity and nerve amplitudes. Incomplete POEMS syndrome of polyneuropathy, hepatomegaly and lymphadenopathy, peripheral edema, thrombocytosis (700 000/μm), and pleural effusion was diagnosed according to Gheradi et al.4 In contrast to other published cases, no monoclonal immunoglobulin was detected (immunofixation electrophoresis of serum and urine, on follow-up free light chains by nephelometry).

Although therapy was started (melphalan, prednisolone), neurological symptoms progressed further. After ineffective tamoxifen medication,5 the patient received 4 cycles of ifosfamide (2 × 3000 mg/m²). Peripheral blood stem cells were collected and the patient underwent high-dose chemotherapy with total body irradiation (12 Gy) and melphalan (140 mg/m²) in April 1996. The posttransplantation course was uneventful except for neutropenic fever and severe mucositis. Six years after transplantation the patient is still in good clinical condition. His neurologic status improved considerably; the patient is able to get up and walk short distances on crutches including going upstairs a few steps. Extensor and flexor muscles of the feet were still plegic. All other muscles of the upper and lower extremities had nearly normal strength. Hypesthesia and pallhypesthesia of the lower extremities had slightly improved. All muscular stretch reflexes were absent. Electroneurographic parameters of nerves of the upper extremities normalized. Some of the sclerotic lesions improved (femura, pelvis), some remained unchanged (skull), and no new osteosclerotic lesions occurred. Thrombocyte counts returned to normal, and peripheral edema disappeared. Observed clinical and laboratory improvements could also have at least partially resulted from the mobilization chemotherapy. After 6 years of follow-up, there are no clinical or biochemical signs of progression.

This report demonstrates that, as shown by Jaccard et al,1autologous stem cell transplantation should be considered in patients with POEMS syndrome. This patient showed a good partial remission now lasting more than 6 years after transplantation.