Oral roflumilast 250 microgram daily will be started at the baseline visit for 4 weeks. For those who can tolerate the initial 4-week treatment, roflumilast will be increased to 500 microgram daily, allowing subsequent dose reduction back to 250 microgram daily in case of CTCAE grade 3 or 4 toxicities.

Non-cystic Fibrosis Bronchiectasis

Roflumilast, a phosphodiesterase 4 (PDE4) inhibitor is approved worldwide (including Hong Kong) for treatment of severe chronic obstructive pulmonary disease (COPD) with frequent exacerbations. Roflumilast has been shown to have anti-inflammatory effect in patients with COPD, with significant reduction of sputum absolute neutrophil count, IL-8 and neutrophil elastase compared with placebo treatment. Roflumilast can also improve the lung function parameters in patients with COPD and reduce the rate of moderate-to-severe exacerbations.

Roflumilast Oral Tablet

Queen Mary Hospital

Roflumilast in Non-CF Bronchiectasis Study (2019)

Anti-inflammatory Effects of Roflumilast Treatment for 12 Weeks in Stable-state Non-cystic Fibrosis Bronchiectasis

Daily sputum volume is determined as the average of a three consecutive day collection (9:00 a.m. to 9:00 a.m.) at home, using clear pre-labeled sterile plastic (60 ml) pots stored at 4°C. Subjects are instructed and trained to completely empty the contents of their mouth before expectorating into the sputum pots to ensure minimal contamination by saliva and food debris. The volume of a 24-hour sputum specimen is determined as the volume of water (to the nearest 0.1 ml) in an adjacent identical pot containing water at the same level as the sputum in the sputum-containing pot.

A fresh sputum sample is collected in a sterile clear plastic pot after thorough mouth emptying on the day of planned clinic visit. Sputum leukocyte density is measured within 2 hours of collection by a designated technician, based on five aliquots chosen randomly from the center of a fresh specimen, which are then serially diluted with phosphate-buffered saline (PBS) and read with a light microscope and a hemocytometer as we previously described.

Fresh sputum sample is stored at −70° C within 15 min of collection until ultracentrifugation (100,000 g for 30 min at 4° C) to obtain the sol phase used for enzyme-linked immunoabsorbent assay of cytokines (IL-1β, IL-8, TNF-alpha, IL-17) and LTB4 levels using commercially available kits.

Sputum neutrophil elastatse is measured using a commercially available kinetic, chromogenic microtitre plate assay with succinyl-Ala-Ala-Pro-Val-p-nitroanalide (Bachem) as a substrate, as we previously described. A standard curve, constructed from known concentrations of purified elastase, is included in each assay.

Sputum for bacterial culture is saved and sputum bacterial load is measured at designated visits as described previously with modifications.

Patients' HRQoL will be measured by St. George's Respiratory Questionnaire Hong Kong Chinese version (SGRQ-HK). SGRQ-HK is a disease-specific instrument designed to measure impact on overall health, daily life, and perceived well-being in patients with obstructive airway disease. It consists of 2 parts with 50 items. It addresses frequency and severity patients' symptoms, as well as activities that cause or are limited by breathlessness. The scores range from 0 to 100, with higher scores indicating more limitations. The minimally important difference is a mean change of 4 units for slightly efficacious treatment, 8 units for moderately efficacious change and 12 units for very efficacious treatment. SGRQ-HK has been validated in patients with bronchiectasis and is considered a valid and sensitive instrument for determining quality of life in bronchiectasis patients.

James Chung-Man HO

Clinical Associate Professor

The University of Hong Kong

3 stratification groups: Group 1: High immunologic risk Patients receiving a ≥ 2nd graft and/or Panel Reactive Antibody ≥ 30% and/or Human Leukocyte Antigen (HLA) mismatches ≥ 4 Group 2: High non-immunologic risk Donors over 60 years of age and/or Donor between 50 to 59 years of age who have died of stroke, or had a history of high blood pressure, or at the time of death had a creatininemia ≥ 135 µmol/L Group 3: Low risk Patients not included in Groups 1 or 2

Renal Transplantation

blood samples

Transplantation Department

Catalytic Antibodies to Predict Uninvasively Late Transplant Failure

Hospices Civils de Lyon

Patients receive PEG-proline-interferon alpha-2b SC on days 1 and 15. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.

Primary Myelofibrosis

Secondary Myelofibrosis

Laboratory Biomarker Analysis

PEG-Proline-Interferon Alfa-2b

Quality-of-Life Assessment

Mayo Clinic in Arizona

P1101 in Treating Patients With Myelofibrosis

Phase II Study of P1101 in Early Myelofibrosis

The proportion of successes will be estimated by the number of successes divided by the total number of evaluable patients. Confidence intervals for the true success proportion will be calculated according to the approach of Duffy and Santner.

The maximum grade for each type of adverse event will be recorded for each patient, and frequency tables will be reviewed to determine patterns. Additionally, the relationship of the adverse event(s) to the study treatment will be taken into consideration.

The distribution of survival time will be estimated using the method of Kaplan-Meier.

Patient-reported symptoms and QOL will be described at each time point using the mean, confidence interval, median, and range. Changes in individual symptoms, changes in a symptom scale composed of symptoms specific to MF patients, and changes in the MPN TSS will be investigated. Graphical procedures will include stream plots of individual patient scores and plots of average values over time. Correlational analyses will be done to determine the relationships among patients-reported symptoms and QOL, as well as with clinical outcomes and clinician-assessed symptoms.

Mayo Clinic

National Cancer Institute (NCI)

Best available therapy, as chosen by the investigator for patients who had been on HU in the 1 Year PROUD-PV study

AOP2014 for those patients who had been on AOP2014 in the PROUD-PV study

Polycythemia Vera

Subjects will continue to receive the dosage which delivers the optimal disease response (hematocrit [Hct]<45%, platelets [PLTs]<400 x 109/L and leukocytes [WBCs]<10 x 109/L), as determined in the PROUD-PV study, preferably at the level of target blood values.

Pegylated-Proline-interferon alpha-2b

Best available therapy as selected by the investigator

Best available therapy (BAT)

LKH Graz

University Hospital Innsbruck

Elisabethinen Hospital Linz

Salzburg Regional Hospital

Hanusch Hospital

Medical University Vienna

Hospital Wels-Grieskirchen

University Multiprofile Hospital for Active Treatment "Sveti Georgi", Plovdiv

Specialized Hospital for Active Treatment of Hematological Diseases

Multiprofile Hospital for Active Treatment "Sveta Marina", Varna

Multiprofile Hospital for Active Treatment - Hristo Botev, Vratsa, First Department of Internal Medicine

University Hospital Brno

University Hospital Hradec Kralove

University Hospital Kralovske Vinohrady

University Hospital Motol

Institute Paoli-Calmettes

Hospital Saint-Louis

Clinical Research Center CIC

Aachen University Hospital, Medical Clinic IV

University Hospital Bonn, Center for Internal Medicine, Medical Clinic and Outpatient Clinic III

University Hospital Carl Gustav Carus, Medical Clinic and Polyclinic I

St Istvan and St Laszlo Hospital of Budapest

University of Debrecen

Bekes County Pandy Kalman Hospital, 1st Department of Medicine, Hematology

Kaposi Mor County Teaching Hospital

University of Szeged, Albert Szent-Gyorgyi Clinical Center, Koranyi fasor 6

University Hospital in Cracow

Independent Public Teaching Hospital No.1 in Lublin

Fryderyk Chopin Provincial Specialized Hospital

Nicolaus Copernicus Municipal Specialist Hospital

Institute of Hematology and Transfusion Medicine

Emergency Clinical County Hospital Brasov

Bucharest University Emergency Hospital

Coltea Clinical Hospital

Baranov Republican Hospital

Samara Kalinin Regional Clinical Hospital

Komi Republican Oncology Center

Tula Regional Clinical Hospital

Yaroslavl Regional Clinical Hospital

University Hospital with Outpatient Clinic F.D. Roosevelt

Saint Cyril and Metod University Hospital Bratislava

Hospital del Mar

Cherkasy Regional Oncology Center, Regional Treatment and Diagnostics Hematology Center

Dnipropetrovsk City Multispecialty Clinical Hospital #4

National Research Center for Radiation Medicine, Institute of Clinical Radiology

Institute of Blood Pathology and Transfusion Medicine

O.F. Herbachevskyi Regional Clinical Hospital

AOP2014 vs. BAT in Patients With Polycythemia Vera Who Previously Participated in the PROUD-PV Study.

An Open-label, Multicenter, Phase IIIb Study Assessing the Long-term Efficacy and Safety of AOP2014 and Standard First Line Treatment (BAT) in Patients With Polycythemia Vera Who Previously Participated in the PROUD-PV Study

The primary efficacy endpoint will be the rate of disease response at assessment visits (every 3 months).
The co-primary efficacy evaluation criterion will be (1) disease response defined as hematologic response: Hct<45% without phlebotomy (at least 3 months since the last phlebotomy), PLTs<400 x 109/L, WBCs<10 x 109/L, and normal spleen size, and (2) disease response defined as hematologic response (Hct<45% without phlebotomy (at least 3 months since the last phlebotomy), PLTs<400 x 109/L, WBCs<10 x 109/L), resolution and/or clinically improvement of disease-related signs (clinical significant splenomegaly) and disease-related symptoms (microvascular disturbances, pruritus, headache).

AOP Orphan Pharmaceuticals AG

PharmaEssentia (Co-Sponsor for USA)

fibrosis

proline

fibrosis/proline

Roflumilast in Non-CF Bronchiectasis Study (2019)

Catalytic Antibodies to Predict Uninvasively Late Transplant Failure

P1101 in Treating Patients With Myelofibrosis

AOP2014 vs. BAT in Patients With Polycythemia Vera Who Previously Participated in the PROUD-PV Study.

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