Neoplasms

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Gland and Hormone Related Diseases

All Conditions

Rare Diseases

Ovarian Neoplasms

Carcinoma, Ovarian Epithelial

Carcinoma

Endocrine Gland Neoplasms

Ovarian Diseases

Adnexal Diseases

Genital Diseases, Female

Genital Diseases

Urogenital Diseases

Female Urogenital Diseases

Pregnancy Complications

Female Urogenital Diseases and Pregnancy Complications

Genital Neoplasms, Female

Urogenital Neoplasms

Endocrine System Diseases

Gonadal Disorders

Neoplasms, Glandular and Epithelial

Neoplasms by Histologic Type

Ovarian Cancer

Ovarian Epithelial Cancer

Antineoplastic Agents

All Drugs and Chemicals

Fluzoparib

Poly(ADP-ribose) Polymerase Inhibitors

Enzyme Inhibitors

patients with Epithelial ovarian cancer, fallopian tube cancer, or primary peritoneal cancer who underwent fluzoparib monotherapy or combination therapy

patients with epithelial ovarian cancer, fallopian tube cancer or primary peritoneal cancer who underwent fluzoparib monotherapy or combination therapy

fluzoparib monotherapy or combination therapy

Qinglei Gao, PhD, MD

Ovarian cancer is one of the most fatal malignant tumors that threaten women's health. The incidence rate is the third place among the female reproductive system malignant tumors, and the mortality rate ranks the first in gynecologic malignancies, the majority of patients have advanced diseases at the time of diagnosis.

This observational study is to evaluate the safety and efficacy of fluzoparib in ovarian cancer patients under real conditions, especially in various subgroups of ovarian cancer patients, in order to provide information about treatment modes for ovarian cancer patients in real-world diagnosis and treatment, and preliminarily evaluate the pharmacoeconomic of fluzoparib in the treatment of ovarian cancer.

Ovarian cancer is one of the most serious malignant tumors that threaten women's health. The incidence rate is third place in the female reproductive system malignant tumors. The mortality rate ranks the first in gynecologic malignancies, and most of them are advanced at the time of diagnosis. The initial treatment of ovarian cancer is mainly surgery and adjuvant chemotherapy. Although most patients can obtain clinical remission after initial treatment, 70% of patients still relapse within 3 years. Therefore, finding effective drugs to prolong the chemotherapy-free interval and exploring comprehensive treatment schemes to prolong survival is the key to treatment.

In recent years, the advent of poly adenosine diphosphate ribose polymerase inhibitors has brought significant changes to the treatment of ovarian cancer. A series of high-level evidence-based medical evidence shows that the application of PARP inhibitors after complete and partial remission of initial treatment or platinum-sensitive recurrence treatment can significantly prolong the progression-free survival time of ovarian cancer patients, Maintenance therapy has become a new model for the treatment of ovarian cancer. At the same time, PARP inhibitors have been approved for posterior line treatment of ovarian cancer. At present, PARP inhibitors have been widely used in clinics and become an important cornerstone of the comprehensive treatment of ovarian cancer.

Fzocus-2 study is a multicenter, randomized, double-blind, placebo-controlled phase III clinical study of fluzoparib in the maintenance treatment of platinum-sensitive recurrent ovarian cancer. The preliminary results were published on the American Society of Gynecological Oncology (SGO) in 2021. A total of 252 patients were included in the study and were treated with fluzoparib (n = 167) or placebo (n = 85). The median follow-up time was 8.5 months. The population-wide data assessed by bIRC showed that the median PFS data were not mature. According to the Kaplan Meier curve, fluzoparib could significantly prolong the PFS (12.9 months: 5.5 months) and reduce the risk of disease progression or death by 75%.

Fzocus-3 is a phase IB study of fluzoparib approved for marketing, which included platinum-sensitive recurrent ovarian cancer patients with germline BRCA mutation who had previously received ≥ 3-line treatment. The objective remission rate of single-drug treatment is as high as 69.9%, and the median progression-free survival time is 12.0 months. It is well comparable with similar drugs and has good safety.

Fluzoparib was approved for the treatment of germline BRCA-mutated (gBRCAm) platinum-sensitive relapsed ovarian cancer in December 2020 and maintenance treatment of platinum-sensitive recurrent ovarian cancer in June 2021. A number of studies on fluzoparib in the field of ovarian cancer are also underway. It is urgent to obtain the real-world data of fluzoparib in the field of ovarian cancer in China. This study will collect information on the baseline characteristics, treatment, and management of ovarian cancer provided in the real world to illustrate the safety and effectiveness of fluzoparib in the treatment of ovarian cancer under the conditions of real clinical practice. It may also include various subgroups of patients with ovarian cancer, which are not involved in the existing phase III registered studies, In order to better understand and explore the use of PARP inhibitors in some patient subgroups and the treatment mode and effect of PARP inhibitors in the treatment of this disease.

HRD score and BRCA gene test should be conducted using blood and tumor samples. serum CA125 levels should also be tested.

A Non-interventional Registry Study of Fluzoparib in the Treatment of Ovarian Cancer

the safety of fluzoparib in ovarian cancer under real-world conditions, especially Incidence of Hematology related adverse reactions ≥ grade 3

the occasional or rare adverse events of fluzoparib in ovarian cancer under real-world conditions

Overall survival, defined as time from first administration fluzoparib to documented death. To assess clinical effectiveness of fluzoparib monotherapy or combination therapy involved in this study by assessment of overall survival (OS) in patients with Ovarian Cancer. Patients will be followed up once 3-6 months at least

Progression-free survival, defined as time from first administration fluzoparib to documented disease progression or death from any cause after baseline, whichever occurs first;. To assess clinical effectiveness of fluzoparib monotherapy or combination therapy involved in this study by assessment of progression free survival (PFS) in patients with Ovarian Cancer. Patients will be followed up once 3-6 months at least.

Objective Response Rate, defined as percentage of patients with complete or partial response according to local assessments. To assess clinical effectiveness of fluzoparib monotherapy or combination therapy involved in this study by assessment of objective response rate (ORR) in patients with Ovarian Cancer. Patients will be followed up once 12 weeks at least.

Disease Control Rate, defined as percentage of patients with complete or partial response or stable disease according to local assessments. To assess clinical effectiveness of fluzoparib monotherapy or combination therapy involved in this study by assessment of disease control rate (DCR) in patients with Ovarian Cancer. Patients will be followed up once 12 weeks at least.

Duration Of Therapy, defined as the time from first administration fluzoparib to the permanent cessation of fluzoparib (including death).

Measurements of tumor biomarkers, such as : CA125, HRD, BRCA, etc.

First Hospital of China Medical University

Cancer Hospital of Xinjiang Medical University

First Affiliated Hospital Xi'an Jiaotong University

Qingyuan City People's Hospital

Zhejiang University

Panzhihua Central Hospital

Liangshan First People's Hospital

Guang'an People's Hospital

Afﬁliated Hospital of North Sichuan Medical College

Cancer Hospital of Guizhou Province

Guizhou International General Hospital

Yichang Central People's Hospital

Wuhan Fourth Hospital

Wuhan Union Hospital, China

Tongji Hospital

Professor

Respiratory Tract (Lung and Bronchial) Diseases

Blood and Lymph Conditions

Immune System Diseases

Carcinoma, Non-Small-Cell Lung

Lung Neoplasms

Lymphoma

Carcinoma, Bronchogenic

Bronchial Neoplasms

Respiratory Tract Neoplasms

Thoracic Neoplasms

Lung Diseases

Respiratory Tract Diseases

Lymphosarcoma

Ensartinib

Protein Kinase Inhibitors

Ensartinib 225 mg administered once daily orally

Yan Xu, Dr.

Mengzhao Wang

The primary objective of this study is to evaluate the efficacy and safety of Ensartinib in advanced ALK-positive non-small cell lung cancer, and the mechanisms of population pharmacokinetics and resistance to Ensartinib.

Participants will receive Ensartinib at 225 mg orally once a day (QD). Treatments will continue until disease progression, meeting one of treatment discontinuation criteria (eg, patient decision, adverse event, pregnancy). At the time of disease progression, participants will enter a survival follow-up until death, withdrawal of consent or study closure, whichever occurs earlier. Collection of venous blood sample from participants included 3 times: before treatment, 8 weeks of treatment, and disease progression. Blood specimens of 8 ml were collected each time for ctDNA NGS testing, and evaluation of Ensartinib population pharmacokinetics with blood sampling after 8 weeks.

A Real World Study of Ensartinib in Advanced ALK-positive NSCLC

A Real World Study of Ensartinib in Advanced ALK-positive Non-small Cell Lung Cancer (NSCLC)

Evaluation of Ensartinib population Cmax pharmacokinetics.

Defined as time from first dose of Ensartinib to disease progression or death due to any causes

ORR per RECIST 1.1 calculated as the proportion of patients with a best overall response defined as complete response (CR) or partial response (PR).

OS, defined as time from first dose of Ensartinib to death due to any cause

Defined as the time from randomization to death from any cause. The OS rate was estimated based on the landmark analysis.

Adverse events are graded according to CTCAE 5.0

Betta Pharmaceuticals Co., Ltd.

Peking Union Medical College Hospital

Chief Director of Department of Respiratory and Critical Care Medicine

Adenocarcinoma, Clear Cell

Adenomyoepithelioma

Adenocarcinoma

Epithelial-myoepithelial Carcinoma

Immune Checkpoint Inhibitors

Antineoplastic Agents, Immunological

physician's choice of chemotherapy for relapsed/refractory ovarian clear cell carcinoma

physician's choice of chemotherapy

immune checkpoint inhibitor based therapy for relapsed/refractory ovarian clear cell carcinoma

immune checkpoint inhibitor based therapy

Qinglei Gao, PhD

The goal of this observational study is to learn about the treatment effectiveness of physician's choice of chemotherapy and the immune checkpoint inhibitor (ICI)-based therapy in patients with relapsed/refractory ovarian clear cell carcinoma (OCCC), and compare the treatment response with the phase II, single-arm clinical trial INOVA to investigate the efficacy of combinational therapy of sintilimab plus bevacizumab. The main questions it aims to answer are:

* What is the efficacy of physician's choice of chemotherapy in relapsed/refractory OCCC patients in the real world?
* Is ICI-based therapy more effective than physician's choice of chemotherapy in real-world for relapsed/refractory OCCC patients?
* Dose the combinational regimens of sintilimab plus bevacizumab in Sintilimab Plus Bevacizumab in Recurrent/Persistent Ovarian Clear Cell Carcinoma (INOVA) trial more effective than physician's choice of chemotherapy?

Participants will be respectively retrieved and extracted de-identified, longitudinal electronic health records (EHR)-derived data.

A Comparative Study of the Treatment of Real-world Patients With Clear Cell Carcinoma of the Ovary

The individual participant data will be available (including data dictionaries). The individual participant data that underlie the results reported in this article after deidentification (text, tables, figures, and appendices) in particular will be shared

The tumor responses to treatment were evaluated by an independent central investigator according to RECIST version 1.1

the time from therapy initiation to the first documented progression or death from any cause, whichever occurred first

the time from therapy initiation to the first documented complete response or partial response

the time from the first documented response to disease progression or death, whichever occurs first

the proportion of patients with complete response, partial response, and stable disease

Henan Cancer Hospital

Qilu Hospital of Shandong University

Hubei Cancer Hospital

Anhui Provincial Cancer Hospital

Peking University Cancer Hospital & Institute

Hunan Province Tumor Hospital

First Affiliated Hospital of Suzhou Medical College

Shandong Tumor Hospital

Chongqing University Cancer Hospital

First Affiliated Hospital of Zhongshan Medical University

Peking University Shenzhen Hospital

Prof.

Symptoms and General Pathology

Syndrome

Preleukemia

Myelodysplastic Syndromes

Anemia

Bone Marrow Diseases

Hematologic Diseases

Precancerous Conditions

Hematinics

Luspatercept

Adult patients (i.e. aged \>=18 years) with diagnosis of MDS according to WHO 2016 classification that met IPSS-R criteria for very low, low, or intermediate-risk MDS treated with Luspatercept

Luspatercept treatment in adults with transfusion-dependent anaemia due to MDS

Matteo Della Porta, MD

Valeria Santini, MD

Lorenza Borin, MD

Daniela Cilloni, MD

Bruno Fattizzo, MD

Pellegrino Musto, MD

Esther Oliva, MD

Marta Riva, MD

Prassede Salutari, MD

Maria Teresa Voso, MD

Myelodysplastic syndromes (MDS) are a group of malignancies characterized by reduced differentiation and increased apoptosis of hematopoietic progenitor cells, leading to ineffective hematopoiesis. Treatment of MDS varies according to prognosis. Patients with low IPSS-R risk have a low probability of progression to acute myeloid leukemia (AML) and the treatment is aimed at controlling cytopenia and improving quality of life (QOL). Anemia is the most common disease feature, occurring in 80%-85% of low-risk patients, 40% of whom eventually become RBC transfusion-dependent (TD).

Luspatercept is a recombinant fusion protein that selectively binds to ligands belonging to the transforming growth factor-beta (TGF-beta) superfamily. Luspatercept binds to GDF11, GDF8, activin B, and other ligands. This binding leads to inhibition of Smad2/3 signaling, which is abnormally high in disease models of ineffective erythropoiesis such as MDS, resulting in erythroid maturation and differentiation.

Luspatercept is now approved for the treatment of adult patients with TD anemia due to very low-, low-, and intermediate-risk MDS with ring sideroblasts, who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy.

FISiM (Fondazione Italiana Sindromi Mielodidplastiche) promotes a multicenter, retrospective observational study to collect information on the efficacy and safety of luspatercept in a real world Italian population of adult patients with transfusion-dependent anemia due to very low- and intermediate-risk MDS with ring sideroblasts

Myelodysplastic syndromes (MDS) are a group of malignancies characterized by reduced differentiation and increased apoptosis of hematopoietic progenitor cells, leading to ineffective hematopoiesis. The incidence of MDS ranges from 1.5 to 4 cases per 100,000 individuals per year. Prognosis is determined by a number of factors, including age, cytogenetic abnormalities, and cytopenia as determined by the Revised International Prognostic Scoring System (IPSS-R), but also by the occurrence of molecular aberrations (eg, gene mutations) and red blood cell (RBC) transfusion dependence.

Treatment of MDS varies according to prognosis. Patients with low IPSS-R risk have a low probability of progression to acute myeloid leukemia (AML) and the treatment is aimed at controlling cytopenia and improving quality of life (QOL), whereas patients with high-risk disease have a shorter life expectancy and treatment is aimed at modifying the natural course of the disease.

Anemia is the most common disease feature, occurring in 80%-85% of low-risk patients, 40% of whom eventually become RBC transfusion-dependent (TD).

Besides lenalidomide, which is exclusively approved for patients with deletion of chromosome 5q, erythropoiesis-stimulating agents (ESAs) constitute the first option for patients with low risk disease. Patients who do not respond to ESAs have very limited options and ultimately require long-term RBC transfusions. Chronic transfusions lead to secondary iron overload and have a deleterious effect on the patient's QOL.

On April 26, 2019, Celgene Europe BV applied for a marketing authorization via the European Medicines Agency (EMA) centralized procedure for luspatercept (trade name Reblozyl). Luspatercept is a recombinant fusion protein that selectively binds to ligands belonging to the transforming growth factor-beta (TGF-beta) superfamily. Luspatercept binds to GDF11, GDF8, activin B, and other ligands. This binding leads to inhibition of Smad2/3 signaling, which is abnormally high in disease models of ineffective erythropoiesis such as MDS, resulting in erythroid maturation and differentiation.

The review of the benefit-risk balance was conducted by the Committee for Medicinal Products for Human Use (CHMP), and the positive opinion was issued on April 30, 2020. The indication approved in the EU is as follows: "Reblozyl is indicated for the treatment of adult patients with TD anemia due to very low-, low-, and intermediate-risk MDS with ring sideroblasts, who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy.

The clinical development program for luspatercept in MDS consists of 3 clinical trials, including the phase 3, randomized, double-blind, placebo-controlled study ACE-536-MDS-001 (MEDALIST) and 2 supportive phase 2, open-label, single-arm trials (A536-03 and A536-05).

In the MEDALIST trial, adult patients with very low, low, or intermediate IPSS-R risk MDS with ring sideroblasts who required RBC transfusions were randomized 2:1 to luspatercept (1mg/kg) or placebo by the SC route every 3 weeks.16 In both treatment groups, best supportive care could be used when clinically indicated, including RBC transfusions or iron chelation therapy, but excluding ESAs or other MDS-directed agents. The primary endpoint of the trial was the proportion of subjects who were RBC transfusion-independent (RBC-TI) over any 56-day period from week 1 to week 24.

In the MEDALIST trial, 229 subjects very low, low or intermediate IPSS-R risk MDS with ring sideroblasts were randomized: 153 to luspatercept and 76 to placebo (ITT population). Forty-nine (21.4%) patients discontinued from the study, with no differences between arms. Patients' baseline characteristics and prior medication use were well balanced across treatment arms. The percentage of responders (RBC-TI during 56 d through week 24) was 37.91% versus 13.16% (P \< 0.0001) for patients receiving luspatercept versus placebo, respectively (Table 1). When the assessment period was extended to 84 days, the proportion of responders was 33.33% versus 11.84% through week 48 and 28.10% versus 7.89% through week 24.

The safety database comprised 571 subjects who received luspatercept. The mean luspatercept treatment duration was 49 weeks (median 45.6). Treatment emergent adverse events (TEAEs) were documented in 95.3% of patients in the pooled luspatercept group, compared to 91.2% of patients in the placebo group. Incidence rates of serious TEAEs (23.8% versus 15.0%), grade ≥3 TEAEs (34.9% versus 25.9%) and TEAEs leading to permanent drug discontinuation (8.8% versus 3.6%) were higher in the pooled luspatercept group. In the MDS cohort, the most frequent TEAEs leading to discontinuation were progression to high-risk MDS, transformation to AML, general physical deterioration and sepsis. Fatal TEAEs were observed in 1.8% versus 2.6% of patients receiving luspatercept versus placebo. The most frequently reported TEAEs (≥15%) in the MDS group were fatigue, diarrhea, nausea, cough, dizziness, hypertension, peripheral edema, headache, viral upper respiratory tract infection, and back pain.

Overall, luspatercept significantly reduced red blood cell (RBC) transfusion requirements in patients with MDS with ring sideroblasts and had a generally manageable tolerability profile in clinical trials. Thus, luspatercept is an emerging treatment option in adults with transfusion-dependent anaemia due to MDS.

Despite that luspatercept is approved based on data of safety and efficacy from a large randomized clinical trial, the authors know that especially for human diseases arising in elderly people, features of subjects included in those randomized trials often do not reflect those of the population in which a therapy is intended to be used. Compliance to treatment and safety in clinical trials can also differ from real world settings. Consequently, in order to drive clinical decision-making process, data from randomized clinical trials should be integrated with evidence generated in a real world setting.

To date, no information is available on the efficacy and safety luspatercept in the treatment of MDS in real world populations. FiSIM (Fondazione Italiana Sindromi Mielodisplastiche) promotes a multicenter, retrospective observational study to collect information on the efficacy and safety of luspatercept in a real world Italian population of adult patients with transfusion-dependent anemia due to very low- and intermediate-risk MDS with ring sideroblasts, who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy.

Efficacy and Safety of Luspatercept: A Study by Fondazione Italiana Sindromi Mielodisplastiche

Efficacy and Safety of Luspatercept in Adult Patients With Transfusion-dependent Anemia Due to Very Low-, Low- and Intermediate-risk (IPSS-R) Myelodysplastic Syndrome With Ring Sideroblasts: a Retrospective Multicenter Study by FISiM-ETS

Percentage of patients who achieved an Hematologic Erythroid Response (HI-E) over any consecutive 56-Day Period.

A modified HI-E response was defined as the percentage of participants meeting the modified HI-E per the International Working Group (IWG 2018) sustained over 56-day consecutive period during the Treatment period. For patients with a baseline RBC transfusion burden of ≥ 4 units/8 weeks, a HI-E is defined as a reduction in RBC transfusion of at least 4 units/8 weeks; for patients with baseline RBC transfusion burden of \<4 units/8 weeks, HI-E is defined as a mean increase in hemoglobin of ≥ 1.5 g/dL for 8 weeks in the absence of RBC transfusions.

Duration of RBC-TI is defined as the longest duration of response for patients who achieved RBC-TI of ≥ 8 weeks during the treatment period Week 1 through Week 24. Patients who maintained RBC-TI through the end of the treatment period were censored at the date of discontinuation or death, whichever occurred first. Median was estimated from unstratified Kaplan Meier method.

Duration of RBC-TI is defined as the longest duration of response for patients who achieved RBC-TI of ≥ 8 weeks during the treatment period Week 1 through Week 48. Patients who maintained RBC-TI through the end of the treatment period were censored at the date of discontinuation or death, whichever occurred first. Median was estimated from unstratified Kaplan Meier method.

Percentage of patients who achieved a Mean Hemoglobin (Hgb) Increase of at Least 1.0 g/dL over any consecutive 56-Day period in absence of RBC Transfusions. A mean hgb increase of ≥ 1.0 g/dL is analyzed as the percentage of patients with a hgb increase ≥1.0 g/dL compared with baseline (after applying the 14/3 day rule) that is sustained over any consecutive 56-day (8-week) period in the absence of RBC transfusions during the treatment period.

Percentage of patients who achieved a Hematologic Improvement in Neutrophil Response (HI-N) Over Any Consecutive 56-day Period. Percentage of patients who achieved a hematologic improvement in neutrophil response (HI-N) per IWG criteria sustained over any consecutive 56-day (8-week) period, during the treatment period (Week 1 to Week 24 and Week 1 to Week 48) HI-N was defined as at least a 100% increase and an absolute increase \> 0.5 X 10\^9/L

Percentage of patients who achieved a Hematologic Improvement in Platelet Response (HI-P) Over Any Consecutive 56-day Period Percentage of patients who achieved a hematologic improvement platelet response (HI-P) was defined as the percentage of participants meeting the HI-P criteria per the IWG sustained over any consecutive 56-day (8-week) period (Week 1 to Week 24 and Week 1 to Week 48) during the treatment period. HI - P response was defined as: Absolute increase of ≥ 30 X 10\^9/L in platelets for participants starting with \> 20 X 10\^9/L platelets; Increase in platelets from \< 20 X 10\^9/L to \> 20 X 10\^9/L and by at least 100%

Percentage of patients who progressed to Acute Myeloid Leukemia (AML). Percentage of participants progressing to AML throughout the course of the study

Time to AML progression is defined as the time between randomization date and the first diagnosis of AML as per World Health Organization (WHO) classification of ≥ 20% blasts in peripheral blood or bone marrow. Participants with a diagnosis of AML were considered to have had an event, participants who did not progress to AML at the time of analysis were censored at the last assessment date which did not indicate progression to AML.

Overall Survival is defined as the time from the date of study drug randomization to death due to any cause. Overall survival was censored at the last date that the participant was known to be alive for participants who were alive at the time of analysis and for those who discontinued from the study or were lost to follow-up.

Survival curves will be estimated by Kaplan Meier Method and differences between groups will be evaluated by the log-rank test. Hazard ratio and their corresponding 95% confidence intervals will be calculated by the Cox Regression Hazard model. All analyses will be performed using SAS version 9.4.

Change From Baseline in Mean Serum Ferritin. Mean change from baseline in mean serum ferritin was calculated as the difference of postbaseline mean serum ferritin (averaged over the specified timepoints) and baseline mean serum ferritin.

Number of patients with Treatment Emergent Adverse Events (TEAEs). The outcome measure describes the number of participants who experienced different types of Treatment-emergent adverse events (TEAEs). TEAEs were defined as Adverse Events (AEs) that started on or after the day of the first dose and on or before 42 days after the last dose of luspatercept. The investigator will determine the relationship of an AE to study drug based on the timing of the AE relative to drug administration and whether or not other drugs, therapeutic interventions, or underlying conditions could provide a sufficient explanation for the event. The severity of an AE is evaluated by the investigator according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) (Version 4.0) where Grade 1 = Mild, Grade 2 = Moderate, Grade 3 = Severe, Grade 4 = Life-threatening and Grade 5 = Death

Mean change in total number of RBC units transfused over a fixed 16-week period (Week 9-24 or Week 33-48) from the total number of RBC units transfused in the 16 weeks immediately on or prior to first dose of study treatment.

Time to Red Blood Cell Transfusion Independence (RBC-TI) - Week 1 Through Week 24. Time to RBC-TI is defined as the time between first dose date and the date of onset of RBC-TI first observed for participants who achieved RBC-TI of ≥ 8 weeks during Week 1 through Week 24

Time to Red Blood Cell Transfusion Independence (RBC-TI) - Week 1 Through Week 48.

Time to RBC-TI is defined as the time between first dose date and the date of onset of RBC-TI first observed for participants who achieved RBC-TI of ≥ 8 weeks during Week 1 through Week 48

Percentage of patients who achieved RBC Transfusion Independence (RBC-TI) ≥ 8 weeks from week 1 to week 24. RBC-TI response is defined as the absence of any RBC transfusion during any consecutive 56-day (8-week) period (ie, Days 1 to 56, Days 2 to 57, Days 3 to 58, etc.) during the first 24 weeks of study treatment. Patients had to have at least 56 days (≥ 8 weeks) of transfusion independence prior to (and including) the Week 24 date to qualify as a responder. Patients who failed to achieve RBC-TI at least 56 days prior to or on the cut-off date were counted as non-responders.

Data will be summarised as frequencies and proportions or as medians and range and differences will be estimated by the chi square test (the Fisher exact test when appropriated) or the t test (the Wilcoxon Mann Whitney U test and the ANOVA model when appropriated based on patient characteristics distribution).

Percentage of patients who achieved RBC-TI ≥ 12 weeks from week 1 to week 24. RBC-TI Response is defined as the absence of any RBC transfusion during any consecutive 84-day (12-week) period (ie, Days 1 to 84, Days 2 to 85, Days 3 to 86, etc.) during the first 24 weeks of treatment.

Data will be summarised as frequencies and proportions or as medians and range and differences will be estimated by the chi square test (the Fisher exact test when appropriated) or the t test (the Wilcoxon Mann Whitney U test and the ANOVA model when appropriated based on patient characteristics distribution).

Percentage of patients who achieved RBC-TI ≥ 12 Weeks from Week 1 to week 48. RBC-TI Response is defined as the absence of any RBC transfusion during any consecutive 84-day (12-week) period (ie, Days 1 to 84, Days 2 to 85, Days 3 to 86, etc.) during the first 48 weeks of treatment.

Data will be summarised as frequencies and proportions or as medians and range and differences will be estimated by the chi square test (the Fisher exact test when appropriated) or the t test (the Wilcoxon Mann Whitney U test and the ANOVA model when appropriated based on patient characteristics distribution).

Percentage of patients who achieved RBC-TI ≥ 8 Weeks from week 1 through week 48.

RBC-TI response is defined as the absence of any RBC transfusion during any consecutive 56-day (8-week) period during the first 48 weeks of study treatment.

Data will be summarised as frequencies and proportions or as medians and range and differences will be estimated by the chi square test (the Fisher exact test when appropriated) or the t test (the Wilcoxon Mann Whitney U test and the ANOVA model when appropriated based on patient characteristics distribution).

Istituto Clinico Humanitas

Fondazione Italiana Sindromi Mielodisplastiche-ETS

Lorlatinib

Juan Li, MD

This study was a multicenter, prospective, non-interventional clinical study that included first-line and late-line patients with advanced non-small cell lung cancer with ALK fusions treated with the third generation ALK-TKI lorlatinib until disease progression, intolerable toxicity, investigator or subject decision to withdraw, lost to follow-up, initiation of other antineoplastic therapy, or death. Clinical pathology including sex, age, ALK mutation status at diagnosis, and clinical stage at diagnosis were collected from medical records. Physical condition as assessed by ECOG-PS before administration of lorlatinib was also recorded. Treatment information was obtained from the records, including dose and timing of ALK-TKI therapy and tumor response, number of prior systemic lines of therapy, and local treatment modalities such as radiotherapy and surgery. Quality of life based on the EORTC QLQ C30+LC29 scale (plus the EORTC QLQ BN20 scale in patients with brain/meningeal metastases) was performed at baseline and at each follow-up point. This study will use REDCap platform to collect and manage the study data information of multi-center patients.

Real World Study of Lolatinib for Advanced ALK+ NSCLC Patients

Real World Study of Lolatinib for Patients With Advanced/Metastatic Anaplastic Lymphoma Kinase (ALK) - Positive Non-Small Cell Lung Cancer (NSCLC)

Intracranial and systemic PFS as assessed by the investigators using RECIST V1.1 or RANO-BM Separately.

Overall survival for first-line and late-line of lorlatinib medication

the Sum of percentage of participants with CR and PR as assessed by RECIST v1.1 or RANO-BM criteria every 8-12 weeks.

Number and percentage of participants with treatment-related AEs as assessed by CTCAE v5.0, and the duration of AEs on every grade.

Change from baseline in quality of life of patients on EORTC QLQ-C30 at every 8-12 Weeks.

Change from baseline in quality of life complemented by EORTC QLQ-LC29 at every 8-12 Weeks.

Change from baseline in activities of daily living of patients with CNS metastases on EORTC QLQ-BM20 at every 8-12 Weeks.

Sichuan Cancer Hospital and Research Institute

Principal Investigator

Immunomodulating Agents

Immunologic Factors

Patients with NSCLC patients receiving neoadjuvant therapy and undergoing surgeries. Resectability after neoadjuvant therapy is judged by the multidisciplinary team of thoracic surgeon, oncologist and radiation oncologist.

Patients with operable stage I-III NSCLC who have previously undergone platinum drugs and immune checkpoint inhibitors(Anti PD-1) and then undergone radical surgery

Neoadjuvant immunochemotherapy

Patients with operable stage I-III NSCLC who have previously undergone platinum-based chemotherapy regimens and then undergone radical surgery

Neoadjuvant chemotherapy

Jie He, Dr.

Despite the clinical success of immune checkpoint blockade (ICB), in neoadjuvant setting, there is still a lack of valid data for operable NSCLC in the real world. This study aim to compare the clinical outcomes (pathologic response rate versus survival) of neoadjuvant immunochemotherapy with neoadjuvant chemotherapy in the real world, to explore the impact of clinicopathological factors on clinical outcomes in neoadjuvant immunochemotherapy setting, and to identify potential neoadjuvant immunochemotherapy beneficiaries.

Although the application of neoadjuvant immunotherapy in NSCLC has brought a new treatment option to many patients, most evidence is based on interventional clinical trials, in which the participants are highly selected and the therapeutic strategies are restricted, resulting in limited representation. There is still a lack of large-scale multicenter real-world data to further verify the benefits in long term overall survival, identify the potential beneficiaries and optimize the therapeutic strategies of neoadjuvant immunochemotherapy.

To meet the forementioned need, the investigators will perform a multi-center retrospective cohort to describe the patterns of neoadjuvant immunochemotherapy use in real world, compare the efficacy of neoadjuvant immunochemotherapy ,and evaluate prognosis of neoadjuvant immunochemotherapy.

Neoadjuvant Treatment Real-world Clinical Outcomes in NSCLC

Neoadjuvant Treatment Real-world Clinical Outcomes in NSCLC: A Retrospective Multi-center Study (NeoR-World)

Defined as the time from the date of randomization until the date of disease recurrence or death (by any cause in the absence of recurrence).

defined as 0% of viable tumor cells in primary tumor and lymph nodes

OS is defined as the time from surgery tiem until death from any cause.

OS is defined as the time from surgery tiem until death from any cause, estimated from a Kaplan Meier plot of OS at the time of the primary analysis.

EFS was defined as time from study inclusion (first dose date) to disease progression, death, or discontinuation of treatment.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
A Non-interventional Registry Study of Fluzoparib in the Treatment of Ovarian Cancer	NCT05206890	Ovarian Cancer	fluzoparib mono...	18 Years -	Tongji Hospital
A Real World Study of Ensartinib in Advanced ALK-positive NSCLC	NCT05498064	Carcinoma, Non-... Anaplastic Lymp... Ensartinib	Ensartinib	18 Years -	Peking Union Medical College Hospital
A Non-interventional Registry Study of Fluzoparib in the Treatment of Ovarian Cancer	NCT05206890	Ovarian Cancer	fluzoparib mono...	18 Years -	Tongji Hospital
A Comparative Study of the Treatment of Real-world Patients With Clear Cell Carcinoma of the Ovary	NCT06279104	Ovarian Clear C...	physician's cho... immune checkpoi...	18 Years - 75 Years	Tongji Hospital
Efficacy and Safety of Luspatercept: A Study by Fondazione Italiana Sindromi Mielodisplastiche	NCT05520749	Myeloid Dysplas...	Luspatercept	18 Years -	Fondazione Italiana Sindromi Mielodisplastiche-ETS
Real World Study of Lolatinib for Advanced ALK+ NSCLC Patients	NCT06361589	ALK-positive No... Real World Stud...	Lorlatinib	18 Years -	Sichuan Cancer Hospital and Research Institute
A Comparative Study of the Treatment of Real-world Patients With Clear Cell Carcinoma of the Ovary	NCT06279104	Ovarian Clear C...	physician's cho... immune checkpoi...	18 Years - 75 Years	Tongji Hospital
Efficacy and Safety of Luspatercept: A Study by Fondazione Italiana Sindromi Mielodisplastiche	NCT05520749	Myeloid Dysplas...	Luspatercept	18 Years -	Fondazione Italiana Sindromi Mielodisplastiche-ETS
Neoadjuvant Treatment Real-world Clinical Outcomes in NSCLC	NCT05974007	Non-Small Cell ...	Neoadjuvant imm... Neoadjuvant che...	18 Years - 85 Years	Cancer Institute and Hospital, Chinese Academy of Medical Sciences
A Comparative Study of the Treatment of Real-world Patients With Clear Cell Carcinoma of the Ovary	NCT06279104	Ovarian Clear C...	physician's cho... immune checkpoi...	18 Years - 75 Years	Tongji Hospital
A Comparative Study of the Treatment of Real-world Patients With Clear Cell Carcinoma of the Ovary	NCT06279104	Ovarian Clear C...	physician's cho... immune checkpoi...	18 Years - 75 Years	Tongji Hospital
Real World Study of Lolatinib for Advanced ALK+ NSCLC Patients	NCT06361589	ALK-positive No... Real World Stud...	Lorlatinib	18 Years -	Sichuan Cancer Hospital and Research Institute

Spots Global Cancer Trial Database for real world study