Hepatic veno-occlusive disease (HVOD) is a serious life-threatening complication of hematopoietic stem cell transplantation (HSCT). Currently, there is no optimal therapeutic strategy and preventive measures are ill-defined. Ursodeoxycholic acid (UA) is well-tolerated oral medication that has been associated with possible benefit as a prophylactic agent. We sought to summarize and quantify the clinical effects of prophylactic UA in the context of HSCT. We undertook a systematic review of studies addressing the use of UA as monotherapy or in combination with other agents in patients undergoing HSCT. The Search Strategy included MEDLINE (1966 to fourth week of March 2006), EMBASE (1980 to fourth week of March 2006), all EBM Reviews (fourth quarter of 2005), Ovid Healthstar (1966 to fourth week of March 2006), and Google Scholar on March 20, 2006. Six studies, 4 randomized clinical trials and 2 historically controlled studies, representing 824 patients were included in the review. Three randomized clinical trials comparing prophylactic UA with no treatment demonstrated reduced proportion of HVOD (relative risk [RR], 0.34; 95% confidence interval [CI], 0.17-0.66). When the analysis was limited to higher-quality studies, the beneficial effect of UA remained significant (RR, 0.36; 95% CI, 0.15-0.90). Transplant-related mortality was also reduced with the prophylactic use of UA (RR, 0.58; 95% CI, 0.35-0.95). UA did not significantly attenuate the outcomes of acute graft-versus-host disease (RR, 0.76; 95% CI, 0.53-1.09), relapse (RR, 0.77; 95% CI, 0.46-1.31), or overall survival (RR, 1.22; 95 % CI, 0.96-1.54). UA appears effective for HVOD prophylaxis in patients undergoing HSCT and should be considered as a prevention strategy by HSCT centers to reduce HVOD.

Autologous hematopoietic cell transplantation (AHCT) as initial therapy of patients with multiple myeloma (MM) improves survival. However, data to support this approach for relapsed/progressive disease after initial AHCT (AHCT1) are limited. Using Center for International Blood and Marrow Transplant Research data, we report the outcomes of 187 patients who underwent a second AHCT (AHCT2) for the treatment of relapsed/progressive MM. Planned tandem AHCT was excluded. Median age at AHCT2 was 59 years (range, 28 to 72), and median patient follow-up was 47 months (range, 3 to 97). Nonrelapse mortality after AHCT2 was 2% at 1 year and 4% at 3 years. Median interval from AHCT1 to relapse/progression was 18 months, and median interval between transplantations was 32 months. After AHCT2, the incidence of relapse/progression at 1 and 3 years was 51% and 82%, respectively. At 3 years after AHCT2, progression-free survival was 13%, and overall survival was 46%. In multivariate analyses, those relapsing ≥36 months after AHCT1 had superior progression-free (P = .045) and overall survival (P = .019). Patients who underwent AHCT2 after 2004 had superior survival (P = .026). AHCT2 is safe and feasible for disease progression after AHCT1. In this retrospective study, individuals relapsing ≥36 months from AHCT1 derived greater benefit from AHCT2 compared with those with a shorter disease-free interval. Storage of an adequate graft before AHCT1 will ensure that the option of a second autologous transplantation is retained for patients with relapsed/progressive MM.

Quiz Ref ID Myasthenia gravis (MG) is an antibody-mediated disease that affects the neuromuscular junction. Despite advances in immune-targeted therapies, a subset of patients have refractory disease with severe or life-threatening symptoms.¹ Quiz Ref ID Autologous hematopoietic stem cell transplant (HSCT) has been used to treat a variety of autoimmune neurologic conditions, including multiple sclerosis, chronic inflammatory demyelinating polyneuropathy, neuromyelitis optica, stiff person syndrome, and others.^2-5 We report our center’s experience using autologous HSCT for 7 patients with severe MG.