Early Weight-Based Aggressive vs. Non-Aggressive Goal-Directed Fluid Resuscitation in the Early Phase of Acute Pancreatitis: An Open-Label Multicenter Randomized Controlled Trial (The WATERFALL Trial), Design, and Rationale

PURPOSE Relapse is a major cause of treatment failure after allogeneic hematopoietic stem-cell transplantation (allo-HSCT) for high-risk acute myeloid leukemia (HR-AML). The aim of this study was to explore the effect of recombinant human granulocyte colony-stimulating factor (rhG-CSF) combined with minimal-dose decitabine (Dec) on the prevention of HR-AML relapse after allo-HSCT. PATIENTS AND METHODS We conducted a phase II, open-label, multicenter, randomized controlled trial. Two hundred four patients with HR-AML who had received allo-HSCT 60-100 days before randomization and who were minimal residual disease negative were randomly assigned 1:1 to either rhG-CSF combined with minimal-dose Dec (G-Dec group: 100 µg/m2 of rhG-CSF on days 0-5 and 5 mg/m2 of Dec on days 1-5) or no intervention (non–G-Dec group). The primary outcome was relapse after transplantation, and the secondary outcomes were chronic graft-versus-host disease (cGVHD), safety of the treatment, and survival. RESULTS The estimated 2-year cumulative incidence of relapse in the G-Dec group was 15.0% (95% CI, 8.0% to 22.1%), compared with 38.3% (95% CI, 28.8% to 47.9%) in the non–G-Dec group ( P < .01), with a hazard ratio (HR) of 0.32 (95% CI, 0.18 to 0.57; P < .01). There was no statistically significant difference between the G-Dec and non–G-Dec groups in the 2-year cumulative incidence of cGVHD without relapse (23.0% [95% CI, 14.7% to 31.3%] and 21.7% [95% CI, 13.6% to 29.7%], respectively; P = .82), with an HR of 1.07 (95% CI, 0.60 to 1.92; P = .81). After rhG-CSF combined with minimal-dose Dec maintenance, increasing numbers of natural killer, CD8+ T, and regulatory T cells were observed. CONCLUSION Our findings suggest that rhG-CSF combined with minimal-dose Dec maintenance after allo-HSCT can reduce the incidence of relapse, accompanied by changes in the number of lymphocyte subtypes.

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Determining the optimal cholecalciferol dosing regimen in children with CKD: a randomized controlled trial

Nephrology Dialysis Transplantation ◽

10.1093/ndt/gfaa369 ◽

2020 ◽

Author(s):

Arpana Iyengar ◽

Nivedita Kamath ◽

Hamsa V Reddy ◽

Jyoti Sharma ◽

Jyoti Singhal ◽

...

Keyword(s):

Randomized Controlled Trial ◽

Controlled Trial ◽

Fibroblast Growth Factor 23 ◽

Glomerular Disease ◽

Dosing Regimen ◽

Open Label ◽

Intensive Phase ◽

Treatment Groups ◽

Randomized Controlled ◽

Multicenter Randomized Controlled Trial

Abstract Background and objectives The optimal treatment regimen for correcting 25-hydroxyvitamin D (25OHD) deficiency in children with chronic kidney disease (CKD) is not known. We compared cholecalciferol dosing regimens for achieving and maintaining 25OHD concentrations ≥30ng/ml in children with CKD stages 2-4. Design An open-label multicenter randomized controlled trial randomized children with 25OHD concentrations<30ng/ml in 1:1:1 to oral cholecalciferol 3,000 IU daily, 25,000 IU weekly, or 100,000 IU monthly for 3-months (maximum 3 intensive courses).In those with 25OHD ≥30ng/ml 1,000IU cholecalciferol daily (maintenance course) was given for up to 9 months. Primary outcome was achieving 25OHD≥30 ng/ml at end of intensive phase treatment. Results 90 children were randomized to daily(n = 30), weekly(n = 29) or monthly(n = 31) treatment groups. At end of intensive phase, 70/90(77.8%) achieved 25OHD ≥30ng/ml; 25OHD concentrations were comparable between groups (median 44.3, 39.4, and 39.3 ng/ml for daily, weekly, and monthly groups respectively; p = 0.24) with no difference between groups for time to achieve 25OHD ≥30ng/ml (p = 0.28). There was no change in calcium, phosphorus, and parathyroid hormone, but fibroblast growth factor 23(p = 0.002) and klotho(p = 0.001) concentrations significantly increased and were comparable in all treatment groups. Irrespective of dosing regimen, children with glomerular disease had 25OHD concentrations lower than non-glomerular disease (25.8 vs 41.8 ng/ml; p = 0.007). One child had 25OHD concentration of 134 ng/ml and 5.5% had hypercalcemia without symptoms of toxicity. Conclusion Intensive treatment with oral cholecalciferol as daily, weekly or monthly regimens achieved similar 25OHD concentrations between treatment groups without toxicity. Children with glomerular disease required higher doses of cholecalciferol compared to those with non-glomerular disease.

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Acupuncture for patients with mild hypertension: study protocol of an open-label multicenter randomized controlled trial

Trials ◽

10.1186/1745-6215-14-380 ◽

2013 ◽

Vol 14 (1) ◽

pp. 380 ◽

Cited By ~ 11

Author(s):

Juan Li ◽

Hui Zheng ◽

Ling Zhao ◽

Ying Li ◽

Yan Zhang ◽

...

Keyword(s):

Randomized Controlled Trial ◽

Study Protocol ◽

Mild Hypertension ◽

Controlled Trial ◽

Open Label ◽

Randomized Controlled ◽

Multicenter Randomized Controlled Trial

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P0630 / #1767: ‘BALANCED CRYSTALLOIDS’ VERSUS ‘NORMAL SALINE’ FOR INITIAL FLUID RESUSCITATION IN CHILDREN WITH SEPTIC SHOCK – A MULTICENTER RANDOMIZED CONTROLLED TRIAL

Pediatric Critical Care Medicine ◽

10.1097/01.pcc.0000740856.22795.9b ◽

2021 ◽

Vol 22 (Supplement 1 3S) ◽

pp. 306-306

Author(s):

J. Sankar ◽

J. Muralidharan ◽

L. Av ◽

R. Rameshkumar ◽

K. N ◽

...

Keyword(s):

Septic Shock ◽

Randomized Controlled Trial ◽

Fluid Resuscitation ◽

Normal Saline ◽

Controlled Trial ◽

Randomized Controlled ◽

Multicenter Randomized Controlled Trial ◽

Balanced Crystalloids

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A multicenter randomized controlled trial to evaluate the efficacy of surgery vs. radiofrequency ablation for small hepatocellular carcinoma (SURF trial).

Journal of Clinical Oncology ◽

10.1200/jco.2019.37.15_suppl.4002 ◽

2019 ◽

Vol 37 (15_suppl) ◽

pp. 4002-4002 ◽

Cited By ~ 10

Author(s):

Namiki Izumi ◽

Kiyoshi Hasegawa ◽

Yujiro Nishioka ◽

Tadatoshi Takayama ◽

Naoki Yamanaka ◽

...

Keyword(s):

Hepatocellular Carcinoma ◽

Randomized Controlled Trial ◽

Radiofrequency Ablation ◽

Early Stage ◽

Controlled Trial ◽

Small Hepatocellular Carcinoma ◽

Open Label ◽

Randomized Controlled ◽

Multicenter Randomized Controlled Trial

4002 Background: Surgery (SUR) and radiofrequency ablation (RFA) are both known to be effective therapy for treating patients with small oligonodular hepatocellular carcinoma (HCC), however there is only insufficient evidence about which therapy is more preferred approach. This randomized controlled trial was designed to prospectively compare the efficacy of SUR and RFA as the first approach to primary HCC. Methods: In this open-label trial undertaken at 49 hospital in Japan, we recruited patients having primary HCC with tumor foci numbering less than 3, each measuring 3 cm or less, Child-Pugh score of 7 or less, ages between 20 and 79 year. Before randomization, technical and liver functional feasibility for both treatment arms were confirmed by joint chart review by surgeons and hepatologists. Patients were then randomly assigned in a 1:1 ratio to undergo SUR or RFA, stratified by age, infection of hepatitis-C virus, number of tumors, tumor size and institution. The primary endpoint was recurrence free survival (RFS) and overall survival (OS). Results: Between April 2009 and August 2015, total 308 patients were enrolled to this trial. Because of ineligibility 15 patients were excluded, therefore 145 patients underwent SUR and 148 patients underwent RFA finally. There was no perioperative mortality. Under the median follow-up of 5 years, the 3-year RFS of patients underwent SUR and RFA was 49.8%, 47.7%, respectively (hazard ration [HR] 0.96, 95% CI 0.72-1.28; p = 0.793). OS will be analyzed and published after two years. Conclusions: SUR and RFA were both safe therapeutic approaches and provided equally RFS for early stage HCC smaller than 3 cm. Clinical trial information: UMIN000001795.

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