3-year safety follow-up of radium-223 dichloride (Ra-223) in patients (Pts) with castration-resistant prostate cancer (CRPC) and symptomatic bone metastases (Mets) from ALSYMPCA.

2015 ◽  
Vol 33 (7_suppl) ◽  
pp. 195-195 ◽  
Author(s):  
Chris Parker ◽  
Nicholas J. Vogelzang ◽  
A. Oliver Sartor ◽  
Robert E. Coleman ◽  
Fang Fang ◽  
...  
Keyword(s):  
Aplastic Anemia ◽  
Safety Profile ◽  
Safety Data ◽  
Bone Cancer ◽  
Myelogenous Leukemia ◽  
Castration Resistant Prostate Cancer ◽  
Radium 223 ◽  
Long Term Follow Up

195 Background: In ALSYMPCA, the first-in-class α-emitter Ra-223 had a highly favorable safety profile and was well tolerated. Safety monitoring of Ra-223 is essential for a complete safety profile. Here are final safety data including long-term follow-up safety 3 years after last pt’s first injection (inj). Methods: Pts received 6 inj and entered designated follow-up from 4 wks after their last inj to 3 years after first inj. Pts were to be evaluated during tx period and 9 follow-up visits. All adverse events (AEs) were collected until 12 wks after last inj; thereafter, only AEs deemed tx-related were collected. Additional long-term safety data were assessed by specific diseases including acute myelogenous leukemia (AML), myelodysplastic syndrome (MDS), aplastic anemia, and new primary cancer in bone or other organs. Results: Safety population (pts receiving ≥ 1 inj) included 901 pts (Ra-223, n = 600; placebo [pbo], n = 301); 572 pts (Ra-223, n = 405; pbo, n = 167) entered follow-up. 60 pts (Ra-223, n = 49; pbo, n = 11) completed all follow-up visits. Overall, 564 (94%) Ra-223 and 292 (97%) pbo pts had ≥ 1 tx-emergent AE (Table). During long-term follow-up, there were no reports of AML, MDS, or new primary bone cancer. New primary cancers in other organs were reported: 2 Ra-223 (1 bladder, 1 lymph node mets), 3 pbo (2 skin, 1 adenocarcinoma rectum and sigmoideum), and 2 pbo cross-over pts (1 skin, 1 meningioma). Aplastic anemia was reported in 1 Ra-223 pt. Conclusions: Ra-223 remained safe and well tolerated 3 years after the last pt’s first inj. No major safety issues were identified during the ALSYMPCA 3-year long-term follow-up. Clinical trial information: NCT00699751. [Table: see text]

1.5-year post-treatment follow-up of radium-223 dichloride (Ra-223) in patients with castration-resistant prostate cancer (CRPC) and bone metastases from the phase 3 ALSYMPCA study.

2014 ◽  
Vol 32 (4_suppl) ◽  
pp. 9-9 ◽  
Author(s):  
Sten Nilsson ◽  
Nicholas J. Vogelzang ◽  
A. Oliver Sartor ◽  
David Bottomley ◽  
Robert E. Coleman ◽  
...  
Keyword(s):  
Bone Metastases ◽  
Safety Data ◽  
Bone Cancer ◽  
Myelogenous Leukemia ◽  
Primary Cancer ◽  
Castration Resistant Prostate Cancer ◽  
Radium 223 ◽  
Safety Population

9 Background: Ra-223 is a first-in-class alpha-emitting pharmaceutical recently Food and Drug Administration approved for treatment (tx) of patients (pts) with CRPC and symptomatic bone metastases (mets). In ALSYMPCA, Ra-223 significantly improved overall survival by 3.6 months versus placebo (pbo) (HR = 0.70; 95% CI, 0.58-0.83; P < 0.001) and was well tolerated. Reported here are long-term safety data ~1.5 years after the last pt’s final injection (inj) from the entire ALSYMPCA safety population. Methods: Eligible pts had progressive CRPC with ≥ 2 symptomatic bone mets and no known visceral mets, were receiving best standard of care, and had received docetaxel or were unfit for or declined docetaxel. Pts were randomized 2:1 to 6 inj of Ra-223 (50 kBq/kg IV; q 4 wk) or matching pbo. Only adverse events (AEs) considered tx related by the investigator were reported during follow-up. Long-term safety data were assessed by specific diseases, including acute myelogenous leukemia, myelodysplastic syndrome, aplastic anemia, primary bone cancer, or primary cancer in other organs. Results: ALSYMPCA safety population included 901 pts (Ra-223, n = 600; pbo, n = 301). Overall, 25 (4%) Ra-223 and 8 (3%) pbo pts had ≥ 1 tx-related AE (Table). During follow-up, primary cancer in other organs was reported in 5 pts (Ra-223, n = 2; pbo, n = 3). Conclusions: Ra-223 is an effective and well-tolerated tx for CRPC with symptomatic bone mets. No major safety issues were identified within ~1.5 years after tx in the ALSYMPCA safety population. Clinical trial information: NCT00699751. [Table: see text]

scholarly journals Nontransplant therapy for bone marrow failure

Hematology ◽  
2016 ◽  
Vol 2016 (1) ◽  
pp. 83-89 ◽  
Author(s):  
Danielle M. Townsley ◽  
Thomas Winkler
Keyword(s):  
Bone Marrow ◽  
Aplastic Anemia ◽  
Bone Marrow Failure ◽  
Clonal Evolution ◽  
Single Agent ◽  
Telomere Attrition ◽  
Long Term Follow Up ◽  
Treatment Naïve

Abstract Nontransplant therapeutic options for acquired and constitutional aplastic anemia have significantly expanded during the last 5 years. In the future, transplant may be required less frequently. That trilineage hematologic responses could be achieved with the single agent eltrombopag in refractory aplastic anemia promotes new interest in growth factors after years of failed trials using other growth factor agents. Preliminary results adding eltrombopag to immunosuppressive therapy are promising, but long-term follow-up data evaluating clonal evolution rates are required before promoting its standard use in treatment-naive disease. Danazol, which is traditionally less preferred for treating cytopenias, is capable of preventing telomere attrition associated with hematologic responses in constitutional bone marrow failure resulting from telomere disease.

THUR 173 Longterms: 10-year experience of fingolimod in RRMS patients

2018 ◽  
Vol 89 (10) ◽  
pp. A22.3-A22
Author(s):  
Cohen Jeffrey ◽  
Tenenbaum Nadia ◽  
Bhatt Alit ◽  
Pimentel Ron ◽  
Kappos Ludwig
Keyword(s):  
Adverse Events ◽  
Safety Profile ◽  
Brain Volume ◽  
Disability Progression ◽  
Open Label ◽  
T2 Lesions ◽  
Long Term Follow Up ◽  
Efficacy Measures

ObjectivesPresent results for up to 10 years of fingolimod treatment in RRMS patients.MethodsLONGTERMS is an open-label, single-arm, extension study evaluating the long-term safety, tolerability and efficacy of fingolimod in patients who previously participated in earlier fingolimod studies. Key efficacy measures: annualised relapse rate (ARR), proportion of patients free of 6 month confirmed disability progression (6 m-CDP), annualised rate of new or newly enlarging T2 lesions (ARneT2), and annualised rate of brain atrophy (ARBA). Safety analyses: adverse events (AEs) and serious AEs (SAEs) frequencies.Results3168 patients were included in the analysis. ARR decreased with longer exposure from 0.26 (Month [M] 0–12) to 0.20 (M0–60) and 0.19 (M0–120). Most patients remained free from 6 m-CDP at M60 (79.3%) and M120 (68.1%). ARneT2 decreased from 1.31 (M0–12) to 0.90 (M0–60), and 0.71 (M0–120). Change in brain volume was stable throughout the study (−0.37 [M12], −0.33 [M60] and −0.32 [M120]). Long-term exposure did not raise new safety concerns. No increase in frequencies of AEs or SAEs per year was observed over long-term fingolimod treatment.ConclusionsLong-term follow-up confirmed the established safety profile of fingolimod. Treatment was associated with a sustained low level of disease activity as expressed by clinical and MRI outcomes.DisclaimerPreviously presented at ECTRIMS 2017

ERDAFITINIB in locally advanced or metastatic urothelial carcinoma (mUC): Long-term outcomes in BLC2001.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. 5015-5015 ◽  
Author(s):  
Arlene O. Siefker-Radtke ◽  
Andrea Necchi ◽  
Se Hoon Park ◽  
Jesús García-Donas ◽  
Robert A Huddart ◽  
...  
Keyword(s):  
Safety Profile ◽  
Objective Response ◽  
Locally Advanced ◽  
Optimal Schedule ◽  
Safety Data ◽  
Serum Phosphate Level ◽  
Primary Analysis ◽  
Platinum Based Chemotherapy

5015 Background: Erdafitinib (JNJ-42756493; ERDA) is the only pan-FGFR kinase inhibitor with US FDA approval for treatment of adults with mUC with susceptible FGFR3/2 alterations (alt) and who progressed on ≥ 1 line of prior platinum-based chemotherapy (chemo). Approval was based on data from the primary analysis of the pivotal BLC2001 trial1. Here we report long-term efficacy and safety data from the 8 mg/d continuous dose regimen in BLC2001. Methods: BLC2001 (NCT02365597) is a global, open-label, phase 2 trial of pts with measurable mUC with prespecified FGFR alt, ECOG 0-2, and progression during/following ≥ 1 line of prior chemo or ≤ 12 mos of (neo)adjuvant chemo, or were cisplatin ineligible, chemo naïve. The optimal schedule of ERDA determined in the initial part of the study was 8 mg/d continuous ERDA in 28-d cycles with uptitration to 9 mg/d (ERD 8 mg UpT) if a protocol-defined target serum phosphate level was not reached and if no significant treatment-related adverse events (TRAEs) occurred. Primary end point was the confirmed objective response rate (ORR=% complete response + % partial response). Key secondary end points were progression-free survival (PFS), duration of response (DOR) and overall survival (OS). Results: Median follow-up for 101 patients treated with ERDA 8 mg UpT was ~24 months. Confirmed ORR was 40%. Median DOR was 5.98 mos; 31% of responders had DOR ≥ 1 yr. Median PFS was 5.52 mos, median OS was 11.3 mos. 12-mos and 24-mos survival rates were 49% and 31%, respectively. Median treatment duration was 5.4 mos. The ERDA safety profile was consistent with the primary analysis. No new TRAEs were seen with longer follow-up. Central serous retinopathy (CSR) events occurred in 27% (27/101) of patients; 85% (23/27) were Grade 1 or 2; dosage was reduced in 13 pts, interrupted for 8, and discontinued for 3. On the data cut-off date, 63% (17/27) had resolved; 60% (6/10) of ongoing CSR events were Grade 1. There were no treatment-related deaths. Conclusions: With a median follow-up of 2 yrs, ERDA in mUC + FGFR alt showed a manageable safety profile and consistent efficacy, with median OS of 11.3 mos. 31% had a DOR ≥12 mos and 31% were alive at 24 mos. ERDA monotherapy vs. immune checkpoint inhibitor (PD-1) or chemo is being further analyzed in a randomized control study (THOR; NCT03390504).Reference: Loriot Y, et al. N Engl J Med. 2019;381:338-48. Clinical trial information: NCT02365597 .

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