Allogeneic stem cell transplantation in AML with t(6;9)(p23;q34);DEK-NUP214 shows a favourable outcome when performed in first complete remission

Acute myeloid leukaemia (AML) with t(6;9)(p23;q34) is a poor-risk entity, commonly associated with FLT3-ITD (internal tandem duplication). Allogeneic stem-cell tranplantation (allo-SCT) is recommended, although studies analysing the outcome of allo-SCT in this setting are lacking. We selected 195 patients with t(6;9) AML, who received a first allo-SCT between 2000 and 2016 from the EBMT (European Society for Blood and Marrow Transplantation) registry. Disease status at time of allo-SCT was the strongest independent prognostic factor, with a two-year leukaemia-free survival and relapse incidence of 57% and 19% in patients in CR1 (first complete remission), 34% and 33% in CR2 (second complete remission), and 24% and 49% in patients not in remission, respectively (P < 0·001). This study, which represents the largest one available in t(6;9) AML, supports the recommendation to submit these patients to allo-SCT in CR1.     

A case series of symptomatic intraluminal duodenal duplication cysts: presentation, endoscopic therapy, and long-term outcome (with video)

BACKGROUND: Duodenal duplication cysts are rare congenital anomalies. Symptomatic cases have classically been treated by surgical resection, which can be complex because of the close proximity of the cysts to the papilla. OBJECTIVE: To describe a series of 8 patients with symptomatic duodenal duplication cysts who were treated endoscopically, with a special focus on the long-term outcome. DESIGN: Retrospective case series. SETTING: Three tertiary-care European academic hospitals. PATIENTS: Eight patients, age 8 to 72 years, were treated endoscopically for symptomatic intraluminal duodenal duplication cysts between 1981 and 2006. Seven patients presented with acute pancreatitis, and one patient presented with jaundice. INTERVENTION: Endoscopic incision and marsupialization of the cysts was performed by using a variety of endoscopic tools (needle-knife and regular sphincterotomes, cystotomes, and polypectomy snares). MAIN OUTCOME MEASUREMENTS: Technical success of endoscopic intervention and long-term clinical recurrence of symptoms. RESULTS: No major complications occurred. All patients remained asymptomatic at a median follow-up of 7.3 years. LIMITATIONS: Retrospective study; the small number of patients. CONCLUSIONS: The endoscopic treatment of symptomatic intraluminal duodenal duplication cysts is a safe and effective technique, with excellent long-term results. It represents a minimally invasive alternative to surgical resection and might be considered the preferred therapeutic modality for these cases.     

Constant vs auto-continuous positive airway pressure in patients with sleep apnea hypopnea syndrome and a high variability in pressure requirement

STUDY OBJECTIVES: Auto-continuous positive airway pressure (CPAP) has been reported to have no more efficacy than constant CPAP in unselected patients with sleep apnea hypopnea syndrome (SAHS). The aim of this study was to evaluate patients judged to be good candidates for auto-CPAP because of a high within-night variability in pressure requirement. DESIGN: Single-blind, randomized, cross-over study (2 x 8 weeks) to compare auto-CPAP with constant CPAP. PATIENTS: Outpatients with moderate-to-severe SAHS attending the chest clinic. INTERVENTIONS: Patients were equipped at home in the auto-CPAP mode (model GK418A; Malinckrodt; Nancy, France), using a 4- to 14-cm H(2)O pressure range. Those individuals having a high within-night variability in pressure requirement, assessed at the end of a 14-day run-in period, were included in the cross-over study. Auto-CPAP was compared with constant CPAP (according to a titration night in the sleep laboratory) in terms of compliance, efficacy on apneas (assessed from the pressure monitor), and sleepiness (assessed on the Epworth sleepiness scale). MEASUREMENTS AND RESULTS: Of 90 consecutive patients with SAHS, 27 patients were selected for a within-night variability in pressure requirement exceeding a given threshold. After completion of the cross-over, 24 patients were evaluable. The median percentage of nights the machine was used was 95.5% (range, 45 to 100%) on constant CPAP, and 96.5% (range, 40 to 100%) on auto-CPAP; the median apnea index recorded by the device was 0.40/h (range, 0 to 2.40/h) on constant CPAP, and 0.45/h (range, 0 to 5.80/h) on auto-CPAP (differences not significant). The mean Epworth sleepiness score was significantly (p < 0.01) lower on auto-CPAP (5.1; SD, 2.8) than on constant CPAP (6.1; SD, 2.8). CONCLUSIONS: In patients selected for a high within-night variability in pressure requirement, auto-CPAP administered via a GK418A device was equivalent to constant CPAP based on a titration night in the sleep laboratory. Subjective ratings for sleepiness were slightly lower on auto-CPAP.     

Magnetic Resonance (MR) Metabolic Imaging in Glioma

This review is focused on describing the use of magnetic resonance (MR) spectroscopy for metabolic imaging of brain tumors. We will first review the MR metabolic imaging findings generated from preclinical models, focusing primarily on in vivo studies, and will then describe the use of metabolic imaging in the clinical setting. We will address relatively well‐established ¹H MRS approaches, as well as ³¹P MRS, ¹³C MRS and emerging hyperpolarized ¹³C MRS methodologies, and will describe the use of metabolic imaging for understanding the basic biology of glioma as well as for improving the characterization and monitoring of brain tumors in the clinic.     

Successful heart transplantation following melphalan plus dexamethasone therapy in systemic AL amyloidosis

Recurrence in the allograft and progression in other organs increase mortality after cardiac transplantation in AL amyloidosis. Survival may be improved after suppression of monoclonal light chain (LC) production following high dose melphalan and autologous stem cell transplantation (HDM/ASCT). However, because of high treatment related mortality, this tandem approach is restricted to few patients without significant extra-cardiac involvement. A diagnosis of systemic AL amyloidosis was established in a 45-year old patient with congestive heart failure related to restrictive cardiomyopathy, nephrotic syndrome, peripheral neuropathy, postural hypotension, macroglossia, and lambda LC monoclonal gammopathy. After melphalan and dexamethasone (M-Dex) therapy, which resulted in 80% reduction of serum free lambda LC, he underwent orthotopic cardiac transplantation. Two years later, he remains in a sustained hematologic remission, with no evidence of allograft or extra-cardiac amyloid accumulation. M-Dex should be considered as an alternative therapy in AL amyloid heart transplant recipients ineligible for HDM/ASCT.