Long-term outcomes after autologous stem cell transplantation for patients with POEMS syndrome (osteosclerotic myeloma): a single-center experience

Abstract

The POEMS syndrome (polyradiculoneuropathy, organomegaly, multiple endocrinopathies, monoclonal protein, skin changes) is a rare disease associated with a plasma cell dyscrasia. Patients with disseminated POEMS can be treated with high-dose therapy and autologous stem cell transplantation (ASCT). While clinical improvement is nearly universal in these patients, the long-term outcomes after transplantation are unclear.

POEMS syndrome: Update on diagnosis, risk‐stratification, and management

“…Six other cases of use with or after other alkylator based therapy yielded one death and 4 patients with improvement [22][23][24][25][26][27]176] High-dose chemotherapy with peripheral blood stem cell transplant can also be quite effective, but selection basis may confound these reports. Case series suggest 100% of patients achieve at least some neurologic improvement [18,54,87,140,[149][150][151][152][153][154][155][156][181][182][183][184][185]. Doses of melphalan ranging from 140 to 200 mg m 22 have been used, with the lower doses used for sicker patients.…”

“…Anecdotally, responses are durable, but relapses have been reported [27,186]. Of the 59 patients with POEMS syndrome treated at the Mayo Clinic Rochester, progression-free survival was 98, 94, and 75% at 1, 2, and 5 years, respectively [184]. Symptomatic progressions were rare, whereas radiographic and VEGF progressions were most common.…”

“…Patients must be followed carefully on a quarterly basis tracking the status of deficits comparing these to baseline (Table II) [184]. VEGF responses may occur as soon as 3 months [155], but they can be delayed.…”

“…Recommendations about how to approach organ response have recently been suggested for the purposes of clinical trials since there are more than two-dozen parameters that can be assessed in a given patient with POEMS syndrome, given the multisystem nature of the disease [184,193]. Alternatively, response criteria for POEMS syndrome could be abridged as follows: (1) hematologic response using a modified amyloid response criteria; (2) VEGF response; (3) and a simplified organ response, which is limited to those systems causing the most morbidity, like peripheral neuropathy assessment, pulmonary function testing (diffusion capacity of carbon monoxide), and extravascular overload (grading ascites and pleural effusion as absent, mild, moderate, or severe).…”

High-dose therapy and autologous stem cell transplantation in patients with POEMS syndrome: a retrospective study of the Plasma Cell Disorder sub-committee of the Chronic Malignancy Working Party of the European Society for Blood & Marrow Transplantation

“…[12][13][14] As with MM, the use of HDT/ASCT in patients with POEMS has yielded encouraging results, though most reported series are small and single center experiences. [15][16][17][18][19][20] We, therefore, sought to characterize the effect of ASCT as therapy for patients with POEMS syndrome, determining the effect on organ-specific dysfunction and delineating key patient-and disease-specific factors predicting for outcome. This multi-center retrospective analysis demonstrates the clinical utility of ASCT when incorporated into the clinical management of this systemic disorder.…”