Safety and efficacy of self-administered inhaled loxapine (ADASUVE) in agitated patients outside the hospital setting: protocol for a phase IV, single-arm, open-label trial

IntroductionThere is a need for fast-acting, non-injection antiagitation treatments that are well tolerated and can be used outside of healthcare facilities. In phase II/III trials, an inhaled formulation of loxapine (ADASUVE®), a well-established, first-generation antipsychotic agent, provided rapid control of mild to moderate agitation in the hospital setting. The present study was designed to investigate the safety and efficacy of inhaled loxapine when self-administered outside the hospital setting.Methods and analysisThis phase IV, multicentre, single-arm, open-label clinical trial is being conducted in five countries in Europe: Spain, Germany, Norway, Romania and Austria. The aim is to include approximately 500 patients with schizophrenia or bipolar disorder who previously received and responded well to inhaled loxapine in the hospital setting. Eligible patients will be followed up for 6 months from baseline. They will be given a 10 mg dose of inhaled loxapine to self-administer outside the hospital setting to treat an agitation episode, should one occur. Patients will also be given a short-acting beta-agonist bronchodilator for treatment of possible severe respiratory side effects. The primary endpoint is incidence of serious adverse events (AEs) and respiratory AEs of special interest related to use of inhaled loxapine outside the hospital setting. Secondary endpoints include incidence of other AEs, Clinical Global Impression-Improvement scores up to 2 hours after self-administration of inhaled loxapine, time to improvement of agitation, patient satisfaction with treatment, treatment outcomes according to agitation severity and concordance between the patient (or a family member/caregiver) and the physician in scoring of agitation severity and the decision to self-administer inhaled loxapine.Ethics and disseminationThe protocol received ethics committee approval in the participating countries between January and August 2016. The results of this study will be disseminated through one or more scientific papers.Trial registration numberEudraCT2015-003331-36;NCT02525991; Pre-results.

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Subcutaneous Abatacept for the Treatment of Rheumatoid Arthritis: Longterm Data from the ACQUIRE Trial

The Journal of Rheumatology ◽

10.3899/jrheum.130112 ◽

2014 ◽

Vol 41 (4) ◽

pp. 629-639 ◽

Cited By ~ 23

Author(s):

Mark C. Genovese ◽

César Pacheco Tena ◽

Arturo Covarrubias ◽

Gustavo Leon ◽

Eduardo Mysler ◽

...

Keyword(s):

Rheumatoid Arthritis ◽

Severe Disease ◽

Incidence Rates ◽

Phase Iii ◽

Inadequate Response ◽

Open Label ◽

Serious Adverse Events ◽

Double Blind ◽

Safety And Efficacy ◽

Link Type

Objective.Assess longterm tolerability, safety, and efficacy of subcutaneous (SC) abatacept (ABA) in methotrexate-refractory patients with rheumatoid arthritis (RA).Methods.The phase III, multinational Abatacept Comparison of Sub[QU]cutaneous Versus Intravenous in Inadequate Responders to MethotrexatE (ACQUIRE) trial comprised a 6-month, randomized, double-blind (DB) period, in which patients received intravenous (IV) or SC ABA, plus MTX, followed by an open-label, longterm extension (LTE), in which patients received SC ABA, 125 mg/week. Safety and efficacy from the LTE (∼3.5 yrs of exposure) are reported.Results.Patients who completed the DB period (1372/1385, 99.1%) entered the LTE; 1134 patients (82.7%) kept taking the treatment at time of reporting. Mean (SD) was 31.9 months (6.8); median (range) exposure was 33.0 (8–44) months. Patients entering the LTE had longstanding, moderate-to-severe disease [mean 7.6 (7.9) yrs and DAS28 (C-reactive protein) 6.2 (0.9)]. Incidence rates (events/100 patient-yrs) were reported for serious adverse events (8.76, 95% CI 7.71, 9.95), infections (44.80, 95% CI 41.76, 48.01), serious infections (1.72, 95% CI 1.30, 2.27), malignancies (1.19, 95% CI 0.86, 1.66), and autoimmune events (1.31, 95% CI 0.95, 1.79). Twenty-seven patients (2%) experienced injection-site reactions; all except 1 were mild. American College of Rheumatology 20, 50, and 70 responses achieved during the DB period were maintained through the LTE, and on Day 981 were 80.2% (95% CI 77.2, 83.2), 63.5% (95% CI 58.2, 68.9), and 39.5% (95% CI 34.0, 44.9) for patients who kept taking SC ABA, and 80.0% (95% CI 77.0, 83.0), 63.2% (95% CI 57.8, 68.7), and 39.2% (95% CI 33.7, 44.7) for those who switched from IV to SC ABA.Conclusion.These findings support SC ABA as a well-tolerated and efficacious longterm treatment for patients with RA and inadequate response to MTX (ClinicalTrials.gov identifier NCT00559585).

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Longterm Safety and Efficacy of Subcutaneous Tocilizumab Monotherapy: Results from the 2-year Open-label Extension of the MUSASHI Study

The Journal of Rheumatology ◽

10.3899/jrheum.140665 ◽

2015 ◽

Vol 42 (5) ◽

pp. 799-809 ◽

Cited By ~ 27

Author(s):

Atsushi Ogata ◽

Koichi Amano ◽

Hiroaki Dobashi ◽

Masayuki Inoo ◽

Tomonori Ishii ◽

...

Keyword(s):

Adverse Events ◽

Disease Activity ◽

Additional Treatment ◽

Joint Disease ◽

Open Label ◽

Serious Adverse Events ◽

Double Blind ◽

Safety And Efficacy ◽

American College Of Rheumatology ◽

Open Label Extension

Objective.To evaluate the longterm safety and efficacy of subcutaneous tocilizumab (TCZ-SC) as monotherapy in patients with rheumatoid arthritis (RA).Methods.Of 346 patients who received 24 weeks of double-blind treatment with either TCZ-SC monotherapy, 162 mg every 2 weeks (q2w); or intravenous TCZ (TCZ-IV) monotherapy, 8 mg/kg every 4 weeks; 319 patients continued to receive TCZ-SC q2w in the 84-week open-label extension (OLE) of the MUSASHI study (JAPICCTI-101117). Efficacy, safety, and immunogenicity were evaluated for all patients treated with TCZ during 108 weeks.Results.The proportions of patients who achieved American College of Rheumatology 20/50/70 responses, low disease activity [28-joint Disease Activity Score (DAS28) ≤ 3.2], or remission (DAS28 < 2.6) at Week 24 were maintained until Week 108. The incidences of adverse events and serious adverse events were 498.3 and 16.9 per 100 patient-years (PY), respectively. The overall safety of TCZ-SC monotherapy was similar to that of TCZ-IV monotherapy. Rates of injection site reactions (ISR) through 108 weeks remained similar to rates through 24 weeks. ISR were mild and did not cause any patient withdrawals. No serious hypersensitivity events (including anaphylactic reactions) occurred. Anti-TCZ antibodies were present in 2.1% of patients treated with TCZ-SC monotherapy.Conclusion.TCZ-SC monotherapy maintained a favorable safety profile and consistent efficacy throughout the 108-week study. Like TCZ-IV, TCZ-SC could provide an additional treatment option for patients with RA.

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Open-label, randomized, multicenter, phase IV trial comparing parenteral nutrition using multi-chamber bags (Eurotubes) versus traditional 2/3-chamber bags in subjects with metastatic or locally advanced inoperable cancer requiring parenteral nutrition: The PEKANNUSS trial of the AIO.

Journal of Clinical Oncology ◽

10.1200/jco.2020.38.15_suppl.tps7086 ◽

2020 ◽

Vol 38 (15_suppl) ◽

pp. TPS7086-TPS7086

Author(s):

Georg Martin Haag ◽

Ralf-Dieter Hofheinz ◽

Jann Arends ◽

Thorsten Oliver Goetze ◽

Ingeborg Roetzer ◽

...

Keyword(s):

Food Intake ◽

Parenteral Nutrition ◽

Care Service ◽

Locally Advanced ◽

Self Administration ◽

Open Label ◽

Increased Risk ◽

Low Glucose ◽

Phase Iv ◽

At Home

TPS7086 Background: Approximately 50% of all cancer subjects suffer from cancer anorexia-cachexia syndrome accompanied by an inadequate food intake and predicting mortality, poor therapeutic response, diminished functional capacity, and reduced QoL. Especially in the advanced stages, parenteral nutrition (PN) is often required and accompanied by an increased risk of blood stream infections associated with increased mortality and other serious medical conditions such as sepsis. Furthermore, the switch from oral food intake to PN changes the patient’s everyday life leading to reduced autonomy and flexibility (e.g. due to dependency on home nursing services). This study aims at evaluating the incidence of catheter-related infections (CRI) and the frequency of self-administered parenteral nutrition at home (HPN) in patients receiving standard PN via A) traditional two- or three-chamber bags (often requiring addition of vitamins and/or medications by home care service) or B). the multi-chamber bags Eurotubes (minimizing additional supplements and enabling self-administration by patients at home). In addition, one group of patients will receive low glucose HPN via Eurotubes to investigate a possible benefit on clinical outcome. Methods: This is an open-label, randomized, multicenter, investigator-initiated, phase IV trial. Overall, 350 patients with inoperable metastatic or locally advanced solid tumors who have an indication for parenteral nutrition will be enrolled. Patients will be randomized 1:1:1 ratio to Arm A (Standard PN using Eurotubes) or Arm B (Standard PN using 2/3-chamber bags), or to Arm A-1 (low glucose using Eurotubes). Patients will be assessed (physical exam, ECOG, weight, QoL, lab tests, AEs, HPN documentation) every 4 weeks during the 12 months HPN treatment period. Co-primary endpoints are incidence of CRI and patient autonomy (rate of self-administered PN at home). Secondary endpoints comprise weight change, change in albumin and CRP levels, overall survival, QoL, and safety. Recruitment has just started; first patient in was on February 5th, 2020. Clinical trial information: NCT04105777 .

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Safety and Efficacy of Daratumumab in Patients with Proliferative GN with Monoclonal Immunoglobulin Deposits

Journal of the American Society of Nephrology ◽

10.1681/asn.2020101541 ◽

2021 ◽

pp. ASN.2020101541

Author(s):

Ladan Zand ◽

S. Vincent Rajkumar ◽

Nelson Leung ◽

Sanjeev Sethi ◽

Mireille El Ters ◽

...

Keyword(s):

Partial Remission ◽

Monoclonal Gammopathy ◽

Complete Response ◽

Clinical Trial Registry ◽

Open Label ◽

Serious Adverse Events ◽

Safety And Efficacy ◽

Phase 2 Trial ◽

Open Label Phase ◽

Registration Number

BackgroundTreatment of proliferative GN with monoclonal Ig deposits (PGNMID) is not established. A monoclonal anti-CD38 antibody (daratumumab) is effective in treating multiple myeloma. Abnormal plasma cell clones may play a role in the pathogenesis of PGNMID.MethodsWe evaluated daratumumab’s safety and efficacy in an open-label, phase 2 trial in 11 adults with PGNMID and one with C3 glomerulopathy (C3G) with monoclonal gammopathy. Patients had an eGFR >20 ml/min per 1.73 m2 and proteinuria >1 g/d. They received daratumumab intravenously (16 mg/kg) once weekly for 8 weeks, and then every other week for eight additional doses. Primary outcome was safety, defined as major infections, grade 3 or 4 anemia, leukopenia, or thrombocytopenia. Secondary outcomes were rate of complete remission (proteinuria <500 mg/d with <15% decline in baseline eGFR) or partial remission (>50% reduction in 24-hour proteinuria with <30% decline in eGFR) and proteinuria at 6 and 12 months.ResultsOne patient with C3G had GN unrelated to the monoclonal gammopathy, and one with PGNMID did not complete the first infusion. Five serious adverse events occurred. During the 12 months of the trial, six of the ten patients with PGNMID who received at least one dose of daratumumab had a partial response, and four had a complete response (an overall response rate of 100%). Three patients experienced relapse, two of whom re-entered partial remission after resuming daratumumab therapy. Proteinuria declined significantly, from a median of 4346 mg/d to 1264 mg/d by 12 months.ConclusionsDaratumumab demonstrated an acceptable safety profile and resulted in significant improvement in proteinuria while stabilizing kidney function in patients with PGNMID, suggesting the drug merits further investigation.Clinical Trial registry name and registration number:Daratumumab in Treatment of PGNMID and C3 GN, NCT03095118

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Comparison of safety and efficacy of dextromethorphan and levocloperastine in treatment of dry cough: a randomized open label phase IV clinical trial

International Journal of Basic & Clinical Pharmacology ◽

10.18203/2319-2003.ijbcp20194272 ◽

2019 ◽

Vol 8 (10) ◽

pp. 2284

Author(s):

Arijit Ghosh

Keyword(s):

Clinical Trial ◽

Selection Criteria ◽

Open Label ◽

Safety And Efficacy ◽

Leicester Cough Questionnaire ◽

Open Label Phase ◽

Group A ◽

Group B ◽

Phase Iv

Background: To compare safety and efficacy of dextromethorphan and levocloperastine in treatment of dry cough.Methods: Patients fulfilling the selection criteria were randomized into two groups. Patients in group A were administered dextromethorphan cough lozenges (5 mg) thrice daily for 7 days. Patients in group B were administered syrup levocloperastine (20 mg/5 ml) 5 ml thrice daily for 7 days. Severity and frequency of cough, and Leicester Cough Questionnaire (LCQ) score were assessed at the end of day 7.Results: Levocloperastine significantly decreased (p<0.5) severity and frequency of cough compared to dextromethorphan at day 7. Levocloperastine also significantly increased LCQ score compared to dextromethorphan at day 7.Conclusions: Levocloperastine is significantly more effective compared to dextromethorphan in treatment of dry cough.

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Safety and Efficacy of Repeat Doses of Allogeneic Mesenchymal Stem Cells in Patients with Age Related Frailty

Innovation in Aging ◽

10.1093/geroni/igab046.3524 ◽

2021 ◽

Vol 5 (Supplement_1) ◽

pp. 989-990

Author(s):

Russell Saltzman ◽

Ivonne Schulman ◽

Aisha Khan ◽

Joshua Hare

Keyword(s):

Stem Cells ◽

Mesenchymal Stem Cells ◽

Negative Effects ◽

Multisystem Disorder ◽

Open Label ◽

Serious Adverse Events ◽

Safety And Efficacy ◽

Intravenous Infusions ◽

Age Related ◽

Post Infusion

Abstract Age-related frailty is a common geriatric condition characterized by a decline in physical and immunological capacity that is associated with depletion of endogenous stem cells and leads to increased vulnerability for adverse health outcomes. Allogeneic mesenchymal stem cells (allo-MSCs) exert immunomodulatory effects and promote tissue repair, which may be able to impede the negative effects of the aging process. The objective of this study was to explore the safety and efficacy of repeated infusions of allo-MSCs in subjects with aging-frailty. Mean age at time of first and second infusions was 75.5 and 77 years of age, respectively. In this open-label clinical trial, 24 participants received two intravenous infusions of allo-MSCs with an average interval of 17.6 months between doses. Safety endpoints included incidence of treatment-emergent serious adverse events (TE-SAEs) within 1-month post-infusion and increase in Panel Reactive Antibodies (PRAs) at 6-months post-infusion. Primary efficacy endpoint was change in 6-minute walk test (6MWT) distance at 6-months post-infusion. No TE-SAEs occurred within 1-month post-infusion. PRAs remained stable throughout the study, indicating no evidence of immune rejection. 6MWT increased by 42 meters after the first infusion (P=0.018). Eighteen months later elevation persisted (P=0.026), but did not increase further after the second infusion. In summary, repeated intravenous infusions of allo-MSCs were safe in participants with age-related frailty and showed remarkable improvement in physical performance. Given the excellent safety and efficacy profiles demonstrated in this study, larger clinical trials are warranted to further quantify the efficacy of repeated dosing of allo-MSCs in this multisystem disorder.

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Safety and Efficacy Study of Subcutaneous Homoharringtonine (SC HHT) in Imatinib (IM)-Resistant Chronic Myeloid Leukemia (CML) with the T315I Mutation - Initial Report of a Phase II Trial.

Blood ◽

10.1182/blood.v110.11.1050.1050 ◽

2007 ◽

Vol 110 (11) ◽

pp. 1050-1050 ◽

Cited By ~ 4

Author(s):

H. Jean Khoury ◽

Mauricette Michallet ◽

Selim Corm ◽

Lydia Roy ◽

Dan Jones ◽

...

Keyword(s):

Phase Ii ◽

Phase Ii Trial ◽

Therapeutic Option ◽

Self Administration ◽

Open Label ◽

Initial Report ◽

Transcript Levels ◽

Safety And Efficacy ◽

Coronary Syndrome ◽

T315i Mutation

Abstract HHT [USAN/INN designation - omacetaxine mepesuccinate] is an ester of cephalotaxine with activity in CML independent from tyrosine kinase (TK) inhibition. HHT is also active against Ba/F3 cell lines transfected with various BCR-ABL kinase domain (KD) mutants, including the T315I mutation. BCR-ABL positive cells with the T315I mutation are resistant, both in vitro and in vivo, to IM, as well as 2nd generation TK inhibitors (TKIs), such as dasatinib, nilotinib and bosutinib. Given the lack of active agents in this unique patient (Pt) population, we are conducting a multicenter open-label phase II trial evaluating safety and efficacy of SC HHT in Pts with IM-resistant T315I+ CML in all phases of the disease. The presence of T315I is confirmed at 2 reference labs. Peripheral blood BCR-ABL transcript levels are determined during HHT therapy by qRT-PCR and BCR-ABL KD mutation analyses. Induction consists of repeated cycles of HHT, 1.25 mg/m2 twice daily SC for 14 consecutive days every (q) 28 days until complete hematologic response (CHR) or hematologic improvement. Pts can receive maintenance HHT, 1.25 mg/m2 twice daily SC for 7 days q 28 days, until progression or for up to 24 mo. To date, 19 Pts with Ph+ CML with T315I have been enrolled, 11 in chronic (CP), 4 in accelerated (AP), 4 in blast (BP) phase. Median age is 56 yrs (23 to 79). Pts have failed a median 2 (1 to 8+) prior therapies, including IM (19), dasatinib (10), nilotinib (8), MK-0457 (3), ara-C (5), anthracyclines (3), interferon (2). Median T315I transcript level at baseline is 50% (20–100%). All Pts have completed at least one induction course of HHT, 10 Pts completed 2 courses, 2 Pts 3, and 1 Pt 4. Five Pts are on maintenance HHT (1–7+ mo). SC HHT is well tolerated; no Pt has discontinued treatment due to toxicity. Hyperglycemia, cardiac arrhythmia, and hypotension have not been reported, and local injection reactions are uncommon. One Pt experienced acute coronary syndrome due to HHT-induced anemia. Cycle 2 treatment delays due to neutropenia and/or thrombocytopenia have occurred in 7 of 14 Pts (1–41 days). 4 Pts have experienced progressive disease [1 in CP (splenomegaly, ↑BCR-ABL), 1 AP, 2 BP] after 1–2 induction cycles and were taken off study. T315I transcript levels are no longer detectable in 1 AP and 4 CP Pts, 2 of whom have achieved CHR (1 recently achieved, 1 maintained 7+ mo). In summary, current data from this phase II trial indicate that HHT has an acceptable safety profile and activity in Pts with IM-resistant CML. The SC route offers a convenient dosing format for self administration. Additional enrollment and longer follow-up will determine whether HHT provides a therapeutic option in highly-resistant CML Pts with T315I BCR-ABL KD mutation who have failed one or more TKIs.

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Long-Term Safety and Efficacy of Lisdexamfetamine Dimesylate in Children and Adolescents with ADHD: A Phase IV, 2-Year, Open-Label Study in Europe

CNS Drugs ◽

10.1007/s40263-017-0443-y ◽

2017 ◽

Vol 31 (7) ◽

pp. 625-638 ◽

Cited By ~ 16

Author(s):

David R. Coghill ◽

Tobias Banaschewski ◽

Peter Nagy ◽

Isabel Hernández Otero ◽

César Soutullo ◽

...

Keyword(s):

Children And Adolescents ◽

Open Label ◽

Lisdexamfetamine Dimesylate ◽

Open Label Study ◽

Label Study ◽

Safety And Efficacy ◽

Phase Iv

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Final results of the PUPs B-LONG study: evaluating safety and efficacy of rFIXFc in previously untreated patients with hemophilia B

Blood Advances ◽

10.1182/bloodadvances.2020004085 ◽

2021 ◽

Vol 5 (13) ◽

pp. 2732-2739

Author(s):

Beatrice Nolan ◽

Anna Klukowska ◽

Amy Shapiro ◽

Antoine Rauch ◽

Michael Recht ◽

...

Keyword(s):

High Titer ◽

Factor Ix ◽

Hemophilia B ◽

Weekly Dose ◽

Open Label ◽

Serious Adverse Events ◽

Safety And Efficacy ◽

End Point ◽

Open Label Phase ◽

Bleeding Episodes

Abstract PUPs B-LONG evaluated the safety and efficacy of recombinant factor IX Fc fusion protein (rFIXFc) in previously untreated patients (PUPs) with hemophilia B. In this open-label, phase 3 study, male PUPs (age <18 years) with hemophilia B (≤2 IU/dL of endogenous factor IX [FIX]) were to receive treatment with rFIXFc. Primary end point was occurrence of inhibitor development, with a secondary end point of annualized bleed rate (ABR). Of 33 patients who received ≥1 dose of rFIXFc, 26 (79%) were age <1 year at study entry and 6 (18%) had a family history of inhibitors. Twenty-eight patients (85%) received prophylaxis; median dosing interval was 7 days, with an average weekly dose of 58 IU/kg. Twenty-seven patients (82%) completed the study. Twenty-one (64%), 26 (79%), and 28 patients (85%) had ≥50, ≥20, and ≥10 exposure days (EDs) to rFIXFc, respectively. One patient (3.03%; 95% confidence interval, 0.08% to 15.76%) developed a low-titer inhibitor after 11 EDs; no high-titer inhibitors were detected. Twenty-three patients (70%) had 58 treatment-emergent serious adverse events; 2 were assessed as related (FIX inhibition and hypersensitivity in 1 patient, resulting in withdrawal). Median ABR was 1.24 (interquartile range, 0.00-2.49) for patients receiving prophylaxis. Most (>85%) bleeding episodes required only 1 infusion for bleed resolution. In this first study reporting results with rFIXFc in pediatric PUPs with hemophilia B, rFIXFc was well tolerated, with the adverse event profile as expected in a pediatric hemophilia population. rFIXFc was effective, both as prophylaxis and in the treatment of bleeding episodes. This trial was registered at www.clinicaltrials.gov as #NCT02234310.

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Apatinib as third-line or beyond therapy in patients with chemotherapy-refractory advanced or metastatic adenocarcinoma of stomach or gastroesophageal junction: An open-label, multicenter, post-marketing phase IV study (Ahead-G201).

Journal of Clinical Oncology ◽

10.1200/jco.2018.36.4_suppl.103 ◽

2018 ◽

Vol 36 (4_suppl) ◽

pp. 103-103

Author(s):

Shukui Qin ◽

Wenying Deng ◽

Lu Wen ◽

Junsheng Wang ◽

Guifang Zhang ◽

...

Keyword(s):

Clinical Trial ◽

Gastroesophageal Junction ◽

Stage Iv ◽

Progression Free Survival ◽

Primary Objective ◽

Phase Iii ◽

Open Label ◽

Safety And Efficacy ◽

Post Marketing ◽

Phase Iv

103 Background: The clinical benefit and safety profile of apatinib in advanced gastric cancer have been established in the randomised controlled phase III clinical trial (J Clin Oncol. 34(13):1448-54). A post-marketing study to confirm the safety and efficacy of apatinib is ongoing in a broad range of patients (pts). Methods: This is a single-arm, open-label, multi-center, Phase IV trial with the target sample size of 2000+ (ClinicalTrials.gov Identifier: NCT02426034). Pts were recruited to receive oral apatinib until disease progression, death or unacceptable toxicity. The primary objective was safety, and the secondary objectives included overall response rate (ORR), disease control rate (DCR), progression free survival (PFS) and overall survival (OS). Results: Herein, we report the preliminary data as documented in the EDC System. As of Jul 10, 2017, 1037 pts were enrolled from 138 hospitals across China. Pts characteristics were: median age 59 yrs, male/female 72.0/28.0%, ECOG PS 0/1/≥2 16.6/66.2/17.2%, stage IV 91.0%. 336 (32.4%) pts interrupted treatment and dose modification occurred in 172 (16.6%) pts (reduction 132/12.7%; rise 87/8.4%). Eventually, the mean dosage was 526.2 mg/d. 652 (62.9%) pts had 4407 drug-related adverse events (DRAEs). Grade ≥3 DRAEs occurred in 300 (28.9%) pts. Severe AEs were reported by 221 (21.3%) pts. The most common DRAEs were proteinuria (19.3%), hypertension (18.8%), leukocyte decrease (16.4%), fatigue (14.2%), platelet decrease (13.6%), hand-foot-skin reaction (11.1%) and neutrophil decrease (10.1%). 820 pts were evaluable for efficacy analysis. The best ORR and DCR were 10.7% and 70.0%, respectively. The median PFS and OS were 4.60 (95%CI, 3.25–4.73) and 6.57 (95%CI, 5.78–7.59) months, respectively. Conclusions: Apatinib monotherapy is effective and has a favorable toxicity profile in real-world clinical setting. The preliminary results of this Phase IV study confirmed the safety and efficacy of apatinib demonstrated in the Phase II and III trials. Updated results will be discussed. Clinical trial information: NCT02426034.

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