Graft-versus-host disease (GVHD) of the gastrointestinal (GI) tract is the main cause of nonrelapse mortality (NRM) after allogeneic hematopoietic cell transplantation. Ann Arbor (AA) scores derived from serum biomarkers at onset of GVHD quantify GI crypt damage; AA2/3 scores correlate with resistance to treatment and higher NRM. We conducted a multicenter, phase 2 study using natalizumab, a humanized monoclonal antibody that blocks T-cell trafficking to the GI tract through the α4 subunit of α4β7 integrin, combined with corticosteroids as primary treatment for patients with new onset AA2/3 GVHD. Seventy-five patients who were evaluable were enrolled and treated; 81% received natalizumab within 2 days of starting corticosteroids. Therapy was well tolerated with no treatment emergent adverse events in >10% of patients. Outcomes for patients treated with natalizumab plus corticosteroids were compared with 150 well-matched controls from the MAGIC database whose primary treatment was corticosteroids alone. There were no significant differences in overall or complete response between patients treated with natalizumab plus corticosteroids and those treated with corticosteroids alone (60% vs 58%; P = .67% and 48% vs 48%; P = 1.0, respectively) including relevant subgroups. There were also no significant differences in NRM or overall survival at 12 months in patients treated with natalizumab plus corticosteroids compared with controls treated with corticosteroids alone (38% vs 39%; P = .80% and 46% vs 54%; P = .48, respectively). In this multicenter biomarker–based phase 2 study, natalizumab combined with corticosteroids failed to improve outcome of patients with newly diagnosed high-risk GVHD. This trial was registered at www.clinicaltrials.gov as # NCT02133924.
Water pipe (WP) smoking has become very popular in European countries. A 27-year-old male patient was referred to our clinic with erythrocytosis of unknown origin. His self-reported history included almost daily WP smoking since the age of 14 years. At presentation haemoglobin, haematocrit (Hct) and carboxy-haemoglobin (CO-Hb) levels were elevated to 19.7 g/dl, 54% and 15.4%, respectively. Erythrocytosis was completely reversible upon cessation of WP smoking. Upon follow-up, haemoglobin, Hct and CO-Hb levels undulated according to the intensity of WP usage. Our report shall raise awareness among physicians for WP smoking as a possible cause of secondary erythrocytosis, particularly among younger adults, and provide guidance for the clinical management.
Bevezetés: A könnyűlánc-amyloidosis immunglobulinok könnyűláncaiból származó fi brilláris anyag extracelluláris lerakódása következtében kialakuló kórkép. Célkitűzés: A szerzők célja a szívérintettség, a kezelés és a myeloma fennál-lásának függvényében a túlélési idők meghatározása.
Prognostic factors in light chain amyloidosisIntroduction: Light chain amyloidosis is characterized by extracellular deposition of a fi brillar material derived from immunglobulin light chain fragments. Aim: The aim of the authors was to assess survival depending on cardiac involvement, therapy, and presence of myeloma. Method: The authors studied a retrospective cohort of 29 patients with light chain amyloidosis (13 kappa, 16 lambda) treated in their institution between 2005 and 2014. Results: Twentyone patients had primary amyloidosis, while 8 had coexisting multiple myeloma. One, two and three or more organs were involved in 4, 8, and 17 patients, respectively. Cardiac involvement (22 cases) inversely correlated with survival. Fifteen (52%) patients received chemotherapy only, while 14 (48%) underwent autologous stem cell transplantation with a median survival of 87 and 11.4 months, respectively. Two patients had heart transplantation and survived 70 and 30 months. Median overall survival was 75.8 months. Conclusions: Cardiac transplantation followed by autologous stem cell transplantation is feasible in selected patients with light chain amyloidosis and heart failure.
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