89 research outputs found
Efficacy and safety of once-weekly and twice-weekly bortezomib in patients with relapsed systemic AL amyloidosis: results of a phase 1/2 study
AbstractThis first prospective phase 2 study of single-agent bortezomib in relapsed primary systemic AL amyloidosis evaluated the recommended (maximum planned) doses identified in phase 1 testing (1.6 mg/m2 once weekly [days 1, 8, 15, and 22; 35-day cycles]; 1.3 mg/m2 twice weekly [days 1, 4, 8, and 11; 21-day cycles]). Among all 70 patients enrolled in the study, 44% had ≥ 3 organs involved, including 73% and 56% with renal and cardiac involvement. In the 1.6 mg/m2 once-weekly and 1.3 mg/m2 twice-weekly groups, the hematologic response rate was 68.8% and 66.7% (37.5% and 24.2% complete responses, respectively); median time to first/best response was 2.1/3.2 and 0.7/1.2 months, and 78.8% and 75.5% had response durations of ≥ 1 year, respectively. One-year hematologic progression-free rates were 72.2% and 74.6%, and 1-year survival rates were 93.8% and 84.0%, respectively. Outcomes appeared similar in patients with cardiac involvement. Among all 70 patients, organ responses included 29% renal and 13% cardiac responses. Rates of grade ≥ 3 toxicities (79% vs 50%) and discontinuations/dose reductions (38%/53% vs 28%/22%) resulting from toxicities appeared higher with 1.3 mg/m2 twice-weekly versus 1.6 mg/m2 once-weekly dosing. Both bortezomib dose schedules represent active, well-tolerated regimens in relapsed AL amyloidosis. This study was registered at www.clinicaltrials.gov as #NCT00298766
Efficacy and safety of once-weekly and twice-weekly bortezomib in patients with relapsed systemic AL amyloidosis: results of a phase 1/2 study
Efficacité de la péfloxacine dans le traitement du syndrome de Schnitzler (Série rétrospective de huit patients)
Le syndrome de Schnitzler est une affection qui associe une urticaire chronique systématique à une IgM monoclonale. Il s'accompagne souvent de fièvre et de douleurs ostéo-articulaires. Son évolution est chronique et marquée par le risque de survenue d'une hémopathie lymphoïde. Son traitement est décevant, symptomatique et toujours incomplet. Il fait généralement appel aux corticoïdes systématiques, générant une corticodépendance fréquente, responsable d'une importante iatrogénie. Jusqu'a présent aucun traitement n'a, à notre connaissance, permis une épargne cortisonique durable. Cette absence de thérapeutique concluante est en partie liée au manque de compréhension de la physiopathologie de la maladie. La constatation, faite par hasard, lors d'un traitement par péfloxacine, d'une réponse clinique complète, associée sevrage des corticoïdes, d'une patiente corticodépendante à haut niveau, nous a conduit à traiter d'autres patients. Sept autres patients atteints du syndrome de Schnitzler ont été traités par 800 à 1200 mg de péfloxacine par jour. Nous avons analysés rétrspectivement leurs observations afin d'évaluer l'éfficacité de ce traitement. Une réponse clinique rapide (souvent inférieure à 24 heures), complète et suspensive, durable, aboutissant à une substantielle épargne cortisonique a été observée chez sept des huit patients. La durée moyenne de suivi sous traitement était de 680 jours (2- 3650), aucun effet secondaire majeur n'est survenu...PARIS7-Villemin (751102101) / SudocPARIS-BIUM (751062103) / SudocSudocFranceF
Optimizing treatment strategies in myeloma cast nephropathy: rationale for a randomized prospective trial.
International audienceRenal failure is a frequent complication of multiple myeloma (MM) that strongly affects patient survival. Although a variety of renal diseases may be observed in MM, myeloma cast nephropathy (MCN), a tubulo-interstitial disorder related to precipitation of a monoclonal light chain (LC) within tubular distal lumens, is the main cause of severe and persistent renal failure. To date, the respective frequency and initial evolution of renal disorders associated with monoclonal LC in MM remain poorly defined. Treatment of MCN relies on urgent symptomatic measures and rapid introduction of chemotherapy to reduce the production of monoclonal LC. The introduction of novel chemotherapy regimens based on the association of bortezomib with dexamethasone is likely to have improved the prognosis of MM patients with renal failure. In addition, the combination of novel agents with efficient removal of circulating LC through high cut-off hemodialysis membrane may further increase renal response rate. However, the impact on patient and renal outcomes of these potential therapeutic advances has not been evaluated in prospective studies. The randomized trials EuLITE in the UK and Germany and MYRE in France should help to answer these issues. MYRE is a randomized controlled phase III trial (NCT01208818) that aims to better define the epidemiology and typology of inaugural renal failure in MM and to optimize therapy of MCN patients with and without dialysis-dependent renal failure
Efficacité et mécanismes d'action des immunoglobulines intraveineuses dans la maladie chronique des agglutinines froides (à propos de 6 cas)
PARIS6-Bibl. St Antoine CHU (751122104) / SudocSudocFranceF
Renal transplantation in light chain amyloidosis: coming out of the cupboard.
International audienc
High-dose therapy and autologous blood stem cell transplantation in POEMS syndrome.
We treated 5 patients with polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome and multifocal bone lesions or diffuse bone marrow plasmacytic infiltration with high-dose therapy (HDT) and autologous blood stem cell transplantation. In all cases, the treatment produced remission of plasma cell proliferation associated with marked improvement in the patients' performance status, neurologic symptoms, and other manifestations of the syndrome. HDT with stem cell support should be investigated further as a therapeutic option in patients with POEMS syndrome and disseminated plasma cell dyscrasia
High dose therapy with autologous stem cell transplantation for solitary bone plasmacytoma complicated by local relapse or isolated distant recurrence
High dose therapy with autologous stem cell transplantation for solitary bone plasmacytoma complicated by local relapse or isolated distant recurrence
We report three patients with solitary bone plasmacytoma (SBP) who developed either local recurrence within the radiotherapy field or an isolated distal recurrence and who were treated with high dose therapy supported by autologous stem cell transplantation. All patients remain without evidence of disease for 4-10 years after the procedure. High dose therapy may be of value and require further study in patients with SBP who develop local or distant failure
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