Activity of pomalidomide in patients with immunoglobulin light-chain amyloidosis

Immunoglobulin light-chain (AL) amyloidosis is a rare, incurable plasma cell disorder. Its therapy has benefited immensely from the expanding drug armamentarium available for multiple myeloma. Pomalidomide in combination with weekly dexamethasone (Pom/dex) is active among patients with relapsed myeloma. In the present study, we explored the Pom/dex combination in patients with previously treated AL. Patients were eligible for this prospective phase 2 trial if they had had at least one prior regimen and if they had reasonably preserved organ function. Patients were treated with oral Pom/dex. Thirty-three patients were enrolled. The median age was 66 years. Median time from diagnosis to on-study was 37 months. Eighty-two percent had cardiac involvement. The confirmed hematologic response rate was 48%, with a median time to response of 1.9 months. Organ improvement was documented in 5 patients. The median overall and progression-free survival rates were 28 and 14 months, respectively; the 1-year overall and progression-free survival rates were 76% and 59%, respectively. There was a discordance between the hematologic response and the N-terminal probrain natriuretic peptide response. The most common grade 3-5 adverse events, regardless of attribution, were neutropenia and fatigue. We conclude that pomalidomide appears to be a valuable drug covering an unmet clinical need in patients with previously treated AL. The trial is registered at www.clinicaltrials.gov as NCT00558896. (Blood. 2012;119(23): 5397-5404

Supplemental table e1_ laboratory results

Laboratory result

Data from: Sporadic late-onset nemaline myopathy: clinical spectrum, survival and treatment outcomes

OBJECTIVES: To describe the clinical phenotype, long-term treatment outcome and overall survival of sporadic late onset nemaline myopathy (SLONM) with or without a monoclonal protein (MP). METHODS: we conducted a retrospective chart review of patients seen between September 2000 and June 2017 and collected clinical, laboratory and survival data. Treatment response was classified as mild, moderate, or marked as adjudged by predefined criteria. RESULTS: We identified 28 patients with SLONM, 17 (61%) had an associated MP. Median age at symptom onset was 62 years. Diagnosis was often delayed by a median of 35 months from symptom onset. There was no difference in clinical or laboratory features between patients with or without MP. Although the majority of patients had proximal or axial weakness at onset, about 18% of patients had atypical presentations. 7/9 (78%) patients receiving intravenous immunoglobulin (IVIG), 6/8 (75%) receiving hematologic therapy as either autologous stem cell transplant (ASCT), or chemotherapy and 1/8 (13%) receiving immunosuppressive therapies responded to treatment (p=0.001). All 3 patients with marked response were treated with IVIG, 2 of them had a MP. The 5-year and 10-year overall survivals from symptom onset were 92% and 68% respectively, with no difference between patients with or without MP. CONCLUSIONS: SLONM has a wide spectrum of clinical presentations. In this contemporary case series, overall survival of patients did not seem to be affected by the presence of a MP. Initial treatment with IVIG is reasonable in all patients, followed by ASCT or chemotherapy as second-line therapy in patients with an associated MP

Supplemental table e2_treatment details

Treatment detail