scholarly journals Applicability of Western protocols in resource‐limited setting: Real‐world data of long‐term outcome of intensive treatment of adult acute myeloid leukaemia in Sri Lanka

eJHaem ◽  
2021 ◽  
Author(s):  
Saman Hewamana ◽  
Lakmali Kandabadage ◽  
Thurairajah Skandarajah ◽  
Natasha Pieris ◽  
Eranga Perera ◽  
...  
Keyword(s):  
Sri Lanka ◽  
Acute Myeloid Leukaemia ◽  
Real World ◽  
Real World Data ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Resource Limited ◽  
Limited Setting ◽  
Acute Myeloid

scholarly journals Rituximab Treatment and Long-term Outcome of Patients With Autoimmune Encephalitis

2021 ◽  
Vol 8 (6) ◽  
pp. e1088
Author(s):  
Franziska S. Thaler ◽  
Luise Zimmermann ◽  
Stefan Kammermeier ◽  
Christine Strippel ◽  
Marius Ringelstein ◽  
...  
Keyword(s):  
Real World ◽  
Independent Living ◽  
Disease Onset ◽  
Autoimmune Encephalitis ◽  
Acid Decarboxylase ◽  
Real World Data ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Score Improvement

Background and ObjectivesTo determine the real-world use of rituximab in autoimmune encephalitis (AE) and to correlate rituximab treatment with the long-term outcome.MethodsPatients with NMDA receptor (NMDAR)-AE, leucine-rich glioma-inactivated-1 (LGI1)- AE, contactin-associated protein-like-2 (CASPR2)-AE, or glutamic acid decarboxylase 65 (GAD65) disease from the GErman Network for Research on AuToimmune Encephalitis who had received at least 1 rituximab dose and a control cohort of non–rituximab-treated patients were analyzed retrospectively.ResultsOf the 358 patients, 163 (46%) received rituximab (NMDAR-AE: 57%, CASPR2-AE: 44%, LGI1-AE: 43%, and GAD65 disease: 37%). Rituximab treatment was initiated significantly earlier in NMDAR- and LGI1-AE (median: 54 and 155 days from disease onset) compared with CASPR2-AE or GAD65 disease (median: 632 and 1,209 days). Modified Rankin Scale (mRS) scores improved significantly in patients with NMDAR-AE, both with and without rituximab treatment. Although being more severely affected at baseline, rituximab-treated patients with NMDAR-AE more frequently reached independent living (mRS score ≤2) (94% vs 88%). In LGI1-AE, rituximab-treated and nontreated patients improved, whereas in CASPR2-AE, only rituximab-treated patients improved significantly. No improvement was observed in patients with GAD65 disease. A significant reduction of the relapse rate was observed in rituximab-treated patients (5% vs 13%). Detection of NMDAR antibodies was significantly associated with mRS score improvement. A favorable outcome was also observed with early treatment initiation.DiscussionWe provide real-world data on immunosuppressive treatments with a focus on rituximab treatment for patients with AE in Germany. We suggest that early and short-term rituximab therapy might be an effective and safe treatment option in most patients with NMDAR-, LGI1-, and CASPR2-AE.Class of EvidenceThis study provides Class IV evidence that rituximab is an effective treatment for some types of AE.

Practice and long-term outcome of unprotected left main PCI: real-world data from a nationwide registry

2021 ◽  
pp. 1-8
Author(s):  
Peter Kayaert ◽  
Mathieu Coeman ◽  
Claude Hanet ◽  
Marc J. Claeys ◽  
Walter Desmet ◽  
...  
Keyword(s):  
Real World ◽  
Left Main ◽  
Real World Data ◽  
Term Outcome ◽  
Long Term Outcome ◽  
World Data ◽  
Nationwide Registry

Proportion of Adult AML Patient Population Receiving Allogeneic Stem Cell Transplantation and Long-Term Outcome: Real World Data From the Swedish National Acute Leukemia Registry.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 2289-2289
Author(s):  
Gunnar Juliusson ◽  
Lars Möllgård ◽  
Sören Lehmann ◽  
Åsa Rangert Derolf ◽  
Ulf Tidefelt ◽  
...  
Keyword(s):  
Acute Leukemia ◽  
Cause Of Death ◽  
Real World ◽  
Outcome Data ◽  
World Population ◽  
Real World Data ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Geographical Regions

Abstract Abstract 2289 Poster Board II-266 Allogeneic transplantation is established post-remission therapy in CR1 for AML patients with high risk features, but not in low-risk disease. However, most have intermediate risk, where indications for SCT are less clear. Most SCT outcome data derive from transplant cohorts, and data on patients studied from diagnosis are scarce. Real world population-based data on the effects of transplant is lacking. We have evaluated the role of transplant for AML in the updated Swedish National Acute Leukemia Registry (Blood 2009;113:4179), which covers 98% of all adult patients nationwide diagnosed 1997-2006 and followed up to 2009, i.e., a median observation on survivors of >6 years. Sweden has a population of 9 million people with full access to high-quality therapy for AML including SCT. Registry data were validated from regional transplant center databases. Of 3878 patients with acute leukemia, 3312 had AML (1669 males and 1643 females), and 457 had ALL; 109 had undifferentiated/unclassified AL. AlloSCT in AML non-APL was performed in 8% of patients aged 60-64 years at diagnosis, and 2% of those aged 65-69 years. We then focused on adult AML non-APL patients <60 years (n=782) of which 38% had alloSCT (n=294), similar in both sexes. Two thirds of the alloSCTs were performed in CR1 (64% for males and 72% for females), 22% in CR2 or later CR, and 10% not in CR. Donors were matched unrelated in 48% of transplants in CR1 and ‘not in CR', and 60% in CR2. Of patients having AlloSCT in CR1, 62% are currently alive, as compared to 50% for CR2 patients, and 31% of others. Corresponding figures for sib and unrelated donors were 65% and 56% in CR1, 50% and 53% in CR2, and 40% and 22% if not in CR. Transplant-reported mortality (TRM), i.e. death in CR after SCT was reported in 13% (with another 6% without reported cause of death). Interestingly, these figures were similar for SCT performed in CR1, CR2 and ‘not in CR'. TRM in CR1 was 10(-12)% with sib donor and 16(-19)% with MUD; in CR2 12(-33)% and 17(-25)%; and if not in CR 13(-20)% and 14(-21)%, respectively; ranges include those with no reported cause of death. Swedish healthcare is divided into six geographical regions, all with full therapeutic options and minimal referral in between regions. We are therefore able to study the influence on outcome in relation to the proportion of transplanted AML patients. One region (#3) performed alloSCT in 63% of AML non-APL patients <60 years, another (#2) in 23%, and the remaining four regions in between 33% and 41%. The corresponding proportions for alloSCT in CR1 were 45% (#3), 16% (#2), and 23 through 28% (others). The main reason for this difference was that alloSCT was frequently performed also in ages 40-59 years in region #3. The 8-year total survival of the overall AML non-APL population <60 years (i.e. including those with no SCT) was 52% in region #3, and ranged from 32% through 39% in the other five regions. This real world report on the use of SCT for AML points at existing regional differences in management, and suggests a possible relation to long-term outcome. Disclosures: Möllgård: Celgene: Research Funding.

scholarly journals Adult-onset sporadic chorea: real-world data from a single-centre retrospective study

2021 ◽  
Author(s):  
Roberta Bovenzi ◽  
Matteo Conti ◽  
Rocco Cerroni ◽  
Mariangela Pierantozzi ◽  
Alessandro Stefani ◽  
...  
Keyword(s):  
Clinical Examination ◽  
Real World ◽  
Laboratory Tests ◽  
Clinical Course ◽  
Diagnostic Workup ◽  
Adult Onset ◽  
Real World Data ◽  
Term Outcome ◽  
Long Term Outcome ◽  
World Data

Abstract Background Adult-onset sporadic chorea includes a wide and heterogeneous group of conditions whose differential diagnosis and treatments are often challenging and extensive. Objectives To analyse retrospectively cases of adult-onset sporadic chorea from a single Italian centre to provide insights for a practical approach in the management of these patients. Methods A total of 11,071 medical charts from a 9-year period (2012–2020) were reviewed, identifying 28 patients with adult-onset sporadic chorea (genetic forms excluded). All available data regarding phenomenology, diagnostic workup, aetiology, treatments, and long-term outcome from this cohort were collected and analysed. Results Adult-onset sporadic chorea occurred more frequently in females and presented with an acute-subacute onset. Cerebrovascular diseases accounted for 68% of aetiology; further causes were structural brain lesions, internal diseases, and other movement disorder syndromes. Clinical course was mild, with spontaneous resolution or minimal disturbances in 82% of cases. Neuroimaging was fundamental to diagnose 76% of adult-onset sporadic chorea, an appropriate clinical examination contributed to the 14% of diagnoses, whereas basic laboratory tests to the 10%. Conclusions Revision of real-world data of adult-onset sporadic chorea patients from a single Italian cohort suggests that an accurate clinical examination, neuroimaging, and routine laboratory tests are useful to identify those cases underlying potentially severe but treatable conditions. Although in the majority of cases adult-onset sporadic chorea has mild clinical course and good response to symptomatic treatments, it is essential to run a fast diagnostic workup.

Long-term outcome of minimally invasive mitral valve annuloplasty in disproportionate mitral regurgitation

2020 ◽  
Vol 41 (Supplement_2) ◽  
Author(s):  
M Albano ◽  
S Nagumo ◽  
M Vanderheyden ◽  
J Bartunek ◽  
C Collet ◽  
...  
Keyword(s):  
Mitral Valve ◽  
Mitral Regurgitation ◽  
Minimally Invasive ◽  
Real World ◽  
Independent Predictor ◽  
Real World Data ◽  
Long Term Outcome ◽  
World Data ◽  
Mitral Valve Annuloplasty

Abstract Background Hypothetical concept of disproportionate secondary mitral regurgitation (SMR) has been recently introduced to facilitate patient's selection for mitral valve intervention. However, real world data validating this concept are unavailable. Purpose To investigate long-term effects of minimally invasive mitral valve annuloplasty (MVA) in patients with disproportionate (dSMR) versus proportionate SMR. Methods The study population consisted of 44 consecutive patients (age 67±9,5 years; 64% males) on guidelines-directed therapy with advanced heart failure (HF), reduced LV ejection fraction (EF) (32±9,7%) and SMR undergoing isolated mini-invasive MVA. Patients with organic mitral regurgitation or concomitant myocardial revascularization were excluded. To assess SMR disproportionality, the PISA-derived effective regurgitant orifice area (EROA) and regurgitant volume (RV) were compared to the estimated EROA and RV by using Gorlin formula and pooled real world data. Results According to EROA, a total of 20 (46%) and 24 (54%) patients, respectively, had dSMR and proportionate SMR (pSMR). According to RV, a total of 17 (39%) had dSMR and 27 (61%) had pSMR. Patients with dSMR showed significantly lower prevalence of male gender and higher prevalence of diabetes mellitus than patients with pSMR (p&lt;0,001). Moreover, we observed smaller LV end-diastolic volume, larger EROA and RV (both p&lt;0,01) and higher LV EF (p=0,02) in the dSMR versus the pSMR group. Other baseline characteristics were similar. During median follow up of 4.39 y (IQR 2,2–9,96y), a total of 25 (56%) patients died from any cause while 21 (47%) individuals were readmitted for worsening HF. Patients with dSMR versus pSMR according to both EROA and RV showed significantly lower rate of HF readmissions (both p&lt;0.05) (Figure 1, 2). In Cox regression analysis combining clinical and imaging parameters, dSMR was the only independent predictor of HF readmissions (HR 0.20, 95% CI 0.07–0.60, p=0.004). In contrast, mortality was similar between dSMR and pSMR (NS) with age as the only independent predictor (HR 1,10; 95% CI 1,03–1,18, p=0,003). Conclusions Minimally invasive MVA is associated with significant reduction of HF readmissions in patients with dSMR versus pSMR while the mortality is similar. This suggests the importance of other parameters, i.e. age and degree of LV remodeling, to guide clinical management in SMR. Funding Acknowledgement Type of funding source: None

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