Every-Three-Week (Q3W) Maintenance Dosing of Epoetin Alfa in Anemic Cancer Patients Receiving Chemotherapy: 60,000 U SC QW to Target Hb 12 g/dl, Followed by 80,000 U SC Q3W.

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 4227-4227 ◽  
Author(s):  
Vernon Montoya ◽  
Denise Williams
Keyword(s):  
Health Care Providers ◽  
Response Rate ◽  
Study Drug ◽  
Maintenance Phase ◽  
Care Providers ◽  
Epoetin Alfa ◽  
Open Label ◽  
Serious Adverse Events ◽  
Initiation Phase ◽  
Secondary Endpoints

Abstract The efficacy of epoetin alfa (EPO) 40,000 U SC once weekly (QW) has been established in clinical studies in anemic patients (pts) with cancer receiving chemotherapy (CT). Higher starting doses of EPO may increase initial hemoglobin (Hb) response and subsequent less frequent maintenance dosing may improve dosing flexibility for pts and health care providers. This ongoing, open-label, multicenter, 24-week (wk) study was designed to evaluate the efficacy and safety of EPO at a starting dose of 60,000 U SC QW to a target Hb of 12 g/dL in the Initiation Phase (IP; maximum of 12 wks), followed by 80,000 U SC every 3 wks (Q3W) in the Maintenance Phase (MP) to maintain Hb in the range 11.5–12.5 g/dL. Eligible pts were ≥18 years with nonmyeloid malignancy, had baseline (BL) Hb <11 g/dL, and were scheduled to receive CT Q3W for ≥15 wks. Pts who achieved Hb ≥12 g/dL during the IP subsequently entered the MP; however, pts did not receive the first Q3W maintenance dose until start of the next Q3W CT cycle. Pts were withdrawn if Hb decreased to <11 g/dL during the MP. The primary endpoint was proportion of pts in the IP who achieved an Hb ≥12 g/dL or an Hb increase ≥2 g/dL from BL. Secondary endpoints included Hb increase ≥1 g/dL but <2.0 g/dL from BL in IP, maintenance of mean Hb 11.5–12.5 g/dL in the MP, maintenance of mean Hb >11.0 g/dL but <11.5 g/dL in MP, Hb over time, and transfusions. All Hb and response rates were independent of RBC transfusion within the previous 28 days. Study enrollment was terminated at 115 pts. In this preliminary analysis, the first 69 pts (mean age, 62 years; 65% women; 87% ECOG 0-1) enrolled and who received ≥1 dose of study drug were evaluable for safety and efficacy. Mean BL Hb was 10.2 ± 0.8 g/dL. The proportion of pts in the IP who achieved an Hb ≥12 g/dL or an Hb increase ≥2 g/dL from BL was 74% (49/69). The proportion of pts in the IP with an Hb increase ≥1 g/dL but <2 g/dL from BL was 9% (6/69). Forty-one (59%) pts entered the MP after a median of 5.0 wks in the IP and spent an average of 7.1 wks in the MP. Of these pts, 33 (80%) maintained an average Hb of 11.5–12.5 g/dL and 3 (7%) maintained an average Hb >11.0 g/dL but <11.5 g/dL over the course of the MP. During the MP, mean Hb was 12.1 ± 0.6 g/dL at entry, 11.8 ± 1.0 g/dL (n=15) after 8 wks of Q3W dosing, and 11.6 ± 1.1 g/dL (n=38) at final value. Eleven (27%) pts were withdrawn during the MP because Hb decreased to <11 g/dL. EPO dose was reduced in 25% (17/69) of pts in the IP and in 39% (16/41) of pts in the MP due to Hb >13 g/dL or Hb increase >1.3-g/dL in 2 wks. Thirty-five percent (24/69) of pts had a dose of EPO held at any time during the study for Hb >13 g/dL or another reason. Five (7%) pts were transfused after study day 28. Twenty-six (38%) pts experienced serious adverse events (AEs). Eight (12%) pts experienced a clinically relevant thrombovascular event. Two (3%) pts discontinued due to AEs. Nine (13%) pts died during the study or within the 90-day follow-up period. These preliminary data suggest that initial dosing of EPO 60,000 U QW is associated with a hematopoietic response rate (Hb ≥12 g/dL or Hb increase ≥2 g/dL from BL) of ~75% in pts with cancer and anemia receiving CT, similar to the response rate reported historically for EPO 40,000 U SC QW with dose escalation to 60,000 U SC QW. In addition, among pts who achieved a target Hb ≥12 g/dL with QW dosing and then received Q3W maintenance dosing, EPO 80,000 U SC Q3W maintained mean Hb above 11 g/dL in >85% of pts.

Maintenance Dosing with Epoetin alfa Every Three Weeks (Q3W) in Anemic Patients with Cancer Receiving Chemotherapy Every Three Weeks: Final Results.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 3555-3555 ◽  
Author(s):  
Vernon P. Montoya ◽  
John Xie ◽  
Richard C. Woodman
Keyword(s):  
Adverse Events ◽  
Maintenance Therapy ◽  
Maintenance Phase ◽  
Epoetin Alfa ◽  
Open Label ◽  
Vascular Events ◽  
Serious Adverse Events ◽  
Initiation Phase ◽  
Multi Center Study ◽  
Hematopoietic Response

Abstract Although epoetin alfa is commonly used weekly for the treatment of chemotherapy (CT)-induced anemia, most CT is given at three week intervals. Therefore, to provide increased patient convenience we examined a dosing regimen that would allow patients to receive epoetin alfa 80,000 U Q3W during maintenance therapy. This open-label, multi-center study had two phases: Initiation Phase (IP) of 60,000 U QW to achieve target hemoglobin (Hb) of 12 g/dL (maximum duration 12 weeks), followed by a Maintenance Phase (MP) of 80,000 U Q3W to maintain Hb between 11.0 and 12.5 g/dL. Only those patients achieving Hb ≥ 12 g/dL in the IP entered the MP; once target Hb was achieved, patients proceeded to MP at the beginning of their next full CT cycle. Patients were withdrawn from the MP if Hb decreased to &lt; 11 g/dL. Doses were held for Hb &gt; 13 g/dL and reduced for rate of rise &gt; 1.3 g/dL/2 weeks. Maximum study duration (IP + MP) was 24 weeks. Patients ≥ 18 years of age with non-myeloid malignancy, baseline Hb ≤ 11 g/dL, and CT planned Q3W for ≥ 15 weeks were enrolled. Endpoints included proportion of patients achieving hematopoietic response in the IP (≥ 2 g/dL Hb increase from baseline or Hb ≥ 12 g/dL) and proportion maintaining mean Hb between 11.0 and 12.5 g/dL in the MP. Also analyzed was the proportion of patients maintaining mean Hb between 11.0 and 13.0 g/dL in the MP. A total of 115 patients were enrolled and received at least one dose of epoetin alfa; mean age was 62.2 ± 11.9 years, 66.1% were female, mean baseline Hb was 10.2 ± 0.8 g/dL, and 86.1% had solid tumors. During the IP, 84 of 115 (73.0%) patients achieved hematopoietic response (≥ 2 g/dL Hb increase from baseline or Hb ≥ 12 g/dL). Seventy-three patients (63.5%) who achieved Hb ≥ 12 g/dL entered the MP after a median of 6.0 weeks in IP and spent a mean of 9.8 weeks in MP. Fifty-four of 73 (74.0%) maintained an average Hb between 11.0 and 12.5 g/dL over the course of the MP; 64 (87.7%) maintained an average Hb between 11.0 and 13.0 g/dL over the course of the MP. Twenty-five (34.2%) patients were withdrawn from the MP because Hb decreased to &lt; 11 g/dL. Of 115 patients dosed in IP, 30 (26.1%) had doses reduced and 31 (27.0%) had doses held; of 73 patients dosed in MP, 9 (7.8%) had doses reduced and 16 (13.9%) had doses held. Fifty-one (44.3%) patients experienced serious adverse events. Eleven (9.6%) patients experienced clinically relevant thrombotic vascular events. Two and 4 patients discontinued due to adverse events in the IP and MP, respectively. Eight (7.0%) patients died during the study or within 30 days after last study dose. Although 60,000 U QW was used during the IP to achieve a HR, 40,000 U QW is the recommended initiation dose of epoetin alfa. This study suggests that epoetin alfa 80,000 U administered every three weeks to patients on a Q3W CT regimen is an effective option for maintenance therapy of CT-induced anemia.

Maintenance Dosing with Epoetin alfa Every Two Weeks (Q2W) in Anemic Patients with Cancer Receiving Chemotherapy Every One or Four Weeks: Final Results.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 3770-3770 ◽  
Author(s):  
Stephanie A. Gregory ◽  
John Xie ◽  
Richard C. Woodman
Keyword(s):  
Adverse Events ◽  
Maintenance Phase ◽  
Epoetin Alfa ◽  
Open Label ◽  
Vascular Events ◽  
Serious Adverse Events ◽  
Initiation Phase ◽  
Two Phases ◽  
Multi Center Study ◽  
Hematopoietic Response

Abstract Epoetin alfa administered weekly is a proven treatment for chemotherapy (CT)-induced anemia. To increase patient convenience we examined a dosing regimen that would allow patients to receive epoetin alfa 60,000 U Q2W during maintenance therapy. This open-label, multi-center study had two phases: Initiation Phase (IP) of 60,000 U once weekly (QW) to achieve a target hemoglobin (Hb) of 12 g/dL (maximum duration 12 weeks), followed by a Maintenance Phase (MP) of 60,000 U Q2W to maintain Hb between 11.0 and 12.5 g/dL. Only those patients achieving Hb ≥ 12 g/dL in the IP entered the MP. Patients were withdrawn from the MP if Hb decreased to &lt; 11 g/dL. Doses were held for Hb &gt; 13 g/dL and reduced for rate of rise of &gt; 1.3 g/dL/2weeks. Maximum study duration (IP + MP) was 24 weeks. Patients ≥ 18 years of age with non-myeloid malignancy, baseline Hb ≤ 11 g/dL, and CT planned QW or every 4 weeks for at least 16 weeks were enrolled. Endpoints included proportion of patients achieving hematopoietic response in the IP (≥ 2 g/dL Hb increase from baseline or Hb ≥ 12 g/dL) and proportion maintaining mean Hb between 11.0 and 12.5 g/dL in the MP. A total of 129 patients were enrolled and received at least one dose of epoetin alfa; mean age was 62.6 ± 13.0 years, 65.9% were female, mean baseline Hb was 10.0 ± 0.9 g/dL, and 92.2% had solid tumors. During the IP, 84 of 129 (65.1%) patients achieved hematopoietic response (≥ 2 g/dL Hb increase from baseline or Hb ≥ 12 g/dL). The eighty-four patients who achieved Hb ≥ 12 g/dL entered the MP after a median of 4.0 weeks in IP; treatment duration in MP was 13.2 weeks on average. Fifty-one of 84 (60.7%) maintained an average Hb between 11.0 and 12.5 g/dL over the course of the MP. Sixteen (19.0%) patients were withdrawn from the MP because Hb decreased to &lt; 11 g/dL. Of 129 patients dosed in IP, 14 (10.9%) had doses reduced and 8 (6.2%) had doses held; of 84 patients dosed in MP, 47 (36.4%) had doses reduced and 53 (41.1%) had doses held. Sixty-five (50.4%) patients experienced serious adverse events. Fifteen (11.6%) patients experienced clinically relevant thrombotic vascular events. Three and 5 patients discontinued due to adverse events in the IP and MP, respectively. Fifteen (11.6%) patients died during the study or within 30 days after last study dose. Although 60,000 U QW was used during the IP to achieve a HR, 40,000 U QW is the recommended initiation dose of epoetin alfa. This study of patients receiving CT every 1 or 4 weeks, demonstrated that the majority of those who achieved a HR are able to maintain an acceptable Hb level with epoetin alfa 60,000 U every two weeks.

Epoetin Alfa 60,000 U SC QW Induction Followed by 60,000 U SC Q2W: Final Study Results of a Novel Treatment Strategy for Chemotherapy-Related Anemia.

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 4226-4226 ◽  
Author(s):  
Michael Messino ◽  
Eric McGary ◽  
Denise Williams
Keyword(s):  
Study Drug ◽  
Epoetin Alfa ◽  
Dosing Regimen ◽  
Open Label ◽  
Final Study ◽  
Transfusion Requirements ◽  
Study Results ◽  
Starting Dose ◽  
Secondary Endpoints ◽  
Tumor Types

Abstract Epoetin alfa (EPO) 40,000–60,000 U administered SC QW has been shown to significantly reduce transfusion utilization, increase Hb, and improve quality of life in anemic patients (pts) with cancer receiving chemotherapy (CT). The use of higher initiation doses of epoetin alfa followed by less frequent maintenance dosing was evaluated to provide convenience and dosing flexibility for both pts and clinicians. This open-label, multicenter, pilot study investigated the efficacy of EPO at a starting dose of 60,000 U SC QW for 4 weeks (wks) followed by 60,000 U SC administered every 2 weeks (Q2W) for up to 12 wks in anemic (Hb&lt;11 g/dL) pts with cancer receiving CT. Pts were excluded for planned radiation therapy, prior treatment with an erythropoietic agent within 3 months, and RBC transfusion within 28 days. The primary endpoint was the proportion of pts with a ≥1-g/dL Hb increase from baseline (BL) with QW dosing. Secondary endpoints included mean time to 1-g/dL increase with QW dosing, the proportion of pts in the Q2W dosing phase with ≥1-g/dL Hb increase above the final Hb or maintenance of ≥ the final Hb achieved with QW dosing, and transfusion requirements. Pts with an Hb increase &lt;1 g/dL with QW dosing or an Hb decrease ≥2 g/dL with Q2W dosing were withdrawn for lack of response. If Hb increased to &gt;13 g/dL or if increase was &gt;1.3 g/dL in 2 wks, EPO was held until Hb was &lt;12 g/dL then resumed at 40,000 U SC QW or Q2W. All 51 pts (mean age 66 y, 63% women, 86% ECOG 0-1) enrolled received at least 1 dose of study drug and were evaluable for efficacy and safety. The most common tumor types were lung and breast. Mean BL Hb was 10.1 ± 0.79 g/dL. The dose of EPO was reduced in 45% of pts overall (24% of pts during QW dosing; 41% of pts during Q2W dosing) and held in 63% of pts overall (20% of pts during QW dosing; 59% of pts during Q2W dosing). Thirty-four (67%) pts achieved ≥1-g/dL Hb increase from BL with QW dosing in a mean period of 16 days. Twenty-nine (57%) out of 51 pts went on to receive Q2W dosing. Of these pts, 3 (10%) pts achieved a ≥1-g/dL Hb increase above the Hb achieved with QW dosing and 9 (31%) pts maintained at least the Hb achieved with QW dosing. The mean change in Hb from BL was 1.6 g/dL (n=41) after 4 wks, 1.7 g/dL (n=27) after 8 wks, and 1.7 g/dL (n=10) after 16 wks. Four (7.8%) pts were transfused during the study. The most commonly reported adverse events (AEs) were nausea, asthenia, and fatigue. Thirteen (26%) pts had ≥1 serious AE. One (2%) pt had a clinically relevant thrombotic vascular event (deep venous thrombosis). Three (6%) pts discontinued due to an AE, 2 of whom subsequently died. The total number of deaths on study was 3 (6%). In conclusion, the majority (67%) of pts responded (Hb increase &gt;1 g/dL) to this initial dosing regimen, which compares favorably with responses seen with a lower starting dose (40,000 U SC QW with dose escalation to 60,000 U SC QW). In addition, pts were able to maintain or extend this response with a 60,000 U SC Q2W dosing regimen thereafter.

scholarly journals Comparative maternal outcomes of oral nifedipine and intravenous labetalol for severe hypertension during pregnancy: an open label randomized controlled trial

2020 ◽  
Vol 9 (7) ◽  
pp. 2847
Author(s):  
Monika Sharma ◽  
Sita Thakur ◽  
Kamal Singh ◽  
Shashank Shekhar
Keyword(s):  
Blood Pressure ◽  
Randomized Controlled Trial ◽  
Health Care Providers ◽  
Severe Hypertension ◽  
Controlled Trial ◽  
Care Providers ◽  
Open Label ◽  
Serious Adverse Events ◽  
Randomized Controlled ◽  
Hypertensive Diseases

Background: Hypertensive diseases are commonly seen during pregnancy and remain one of the leading causes of maternal morbidity and mortality. Mostly commonly preferred drugs by health care providers for treatment of severe hypertension during pregnancy are labetalol and hydralazine. However, they require proper storage, intravenous access, and adequately trained staff for usage. Oral nifedipine in contrast is easier to use and widely available.  Objective of this study was to report the efficacy and safety of oral nifedipine as compared to intravenous labetalol for treatment of severe hypertension during pregnancy.Methods: It was an open label randomized controlled trial in which 100 women with severe hypertension during pregnancy were enrolled. They were randomized to receive either incremental doses of intravenous labetalol every 20 minutes (total 300 mg) or 10 mg oral nifedipine every 20 minutes (up to 50 mg) to lower the blood pressure to safer levels.Results: Women receiving oral nifedipine took significantly less time to achieve target blood pressure [(37.6±23.3) minutes (SD) as compared to those receiving intravenous labetalol (52.0 minutes±27.95 (SD)]. Women receiving nifedipine for treatment also required significantly lesser doses to control the blood pressure [mean dose 1.8±1.1 (SD) versus 2.6±1.2 (SD) p=0.006]. There were two failures in labetalol group and one failure in nifedipine group. No serious adverse events were reported in either group.Conclusions: Oral nifedipine is equally efficacious to I.V. labetalol for treatment of severe hypertension during pregnancy and is easier to use in low resource settings.

Randomized Comparison of Every-2-Week Darbepoetin Alfa and Weekly Epoetin Alfa for the Treatment of Chemotherapy-Induced Anemia: The 20030125 Study Group Trial

2006 ◽  
Vol 24 (15) ◽  
pp. 2290-2297 ◽  
Author(s):  
John Glaspy ◽  
Saroj Vadhan-Raj ◽  
Ravi Patel ◽  
Linda Bosserman ◽  
Eddie Hu ◽  
...  
Keyword(s):  
Health Care Providers ◽  
Darbepoetin Alfa ◽  
Study Drug ◽  
The United States ◽  
Phase Iii ◽  
Sensitivity Analyses ◽  
Comparable Efficacy ◽  
Care Providers ◽  
Epoetin Alfa ◽  
Noninferiority Margin

Purpose Chemotherapy-induced anemia is widely treated in the United States with darbepoetin alfa (DA) or epoetin alfa (EA). This noninferiority study systematically compares efficacy and safety of DA and EA using common doses and schedules used in clinical practice. Methods Patients had a diagnosis of nonmyeloid malignancy with ≥ 8 weeks of planned chemotherapy, age ≥ 18 years, and anemia (hemoglobin ≤ 11 g/dL). Patients were randomly assigned 1:1 to DA 200 μg every two weeks (Q2W) or EA 40,000 units every week (QW) for up to 16 weeks with identical dose adjustment rules. Efficacy was assessed by the incidence of RBC transfusion (Kaplan-Meier estimate). The definition of noninferiority was that the upper 95% CI limit of the observed difference in RBC transfusions between groups was less than 11.5%; this noninferiority margin was based on the treatment effect observed in placebo-controlled EA studies. Results Of 1,220 patients randomly assigned, 1,209 received ≥ one dose of the study drug. Common tumor types were lung (26%), breast (21%), and gastrointestinal (18%). Transfusion incidence from week 5 to the end of the treatment phase (the primary end point) was 21% in the DA group and 16% in the EA group; noninferiority was concluded because the upper 95% CI limit of the difference between groups (10.8%) was below the prespecified noninferiority margin. Sensitivity analyses using alternate statistical methods and analysis sets yielded similar results. Hemoglobin, quality of life, and safety end points further support equivalency of the erythropoietic therapies. Conclusion This large, phase III study demonstrates comparable efficacy of DA Q2W and EA QW. Less frequent dosing offers potential benefits for patients, caregivers and health care providers.

Phase 3 trial of momelotinib (MMB) vs ruxolitinib (RUX) in JAK inhibitor (JAKi) naive patients with myelofibrosis (MF).

2017 ◽  
Vol 35 (15_suppl) ◽  
pp. 7000-7000 ◽  
Author(s):  
Ruben A. Mesa ◽  
Jean-Jacques Kiladjian ◽  
John V. Catalano ◽  
Timothy Devos ◽  
Miklos Egyed ◽  
...  
Keyword(s):  
Response Rate ◽  
Transfusion Requirement ◽  
Study Drug ◽  
Spleen Volume ◽  
Open Label ◽  
Phase 3 ◽  
Splenic Volume ◽  
Associated Symptoms ◽  
Symptom Response ◽  
Secondary Endpoints

7000 Background: MMB, an oral JAKi, has been shown in early trials to reduce spleen volume, improve disease associated symptoms (Sx) and improve RBC transfusion (Tx) requirements in patients (pts) with MF. This study was designed to test non-inferiority of MMB vs RUX in splenic volume reduction and Sx amelioration, and superiority in Tx requirement, in JAKi naïve MF pts. Methods: Eligibility: MF, IPSS high risk, Int-2, or symptomatic Int-1; palpable spleen ≥5cm; platelets ≥ 50 K/μl, and no Gr ≥2 peripheral neuropathy (PN). Stratification by Tx dependency and platelets (<100, 100-200 and >200 K/μl). Pts were randomized 1:1 to 24 wks of MMB 200 mg qd + RUX placebo or RUX 20 mg bid (or modified per label) + MMB placebo, after which all pts could receive open label MMB. Assessments: spleen volume by MRI, and pt reported Sx using a daily eDiary of modified MPN-SAF Total Sx Score (TSS). Primary endpoint was splenic response rate (SRR; ≥35% reduction in volume from baseline) at 24 wks. Secondary endpoints, evaluated sequentially at 24 wks, were rates of TSS response (≥50% reduction from baseline), RBC Tx independence (TI), RBC Tx dependence (TD) and of RBC Tx . Results: 175 of 215 (81%) and 201 of 217 (93%) pts randomized to MMB and RUX, respectively, completed the 24 wk DB phase. Efficacy results are shown in Table. Most common Gr ≥3 AEs in the DB phase with MMB were thrombocytopenia (7%) and anemia (6%), and with RUX were anemia (23%), thrombocytopenia (5%) and neutropenia (5%). Gr ≥3 infections occurred in 7% of MMB and 3% of RUX pts. Treatment emergent PN occurred in 22 (10%) of MMB (all Gr ≤2) and 10 (5%) of RUX (9 Gr ≤2, 1 Gr 3) pts in DB phase, none discontinuing study drug for PN. Overall, AEs led to study drug D/C in 13% of MMB and 6% of RUX pts in DB phase. Conclusions: In pts with JAKi naive MF, 24 weeks of MMB is non-inferior to RUX for spleen response but not for symptom response. MMB treatment is associated with a reduced transfusion requirement. NCT01969838. [Table: see text]

scholarly journals Parental Engagement of a Prototype Electronic Diary in an Ambulatory Setting Following Adenotonsillectomy in Children: A Prospective Cohort Study

Children ◽  
2021 ◽  
Vol 8 (7) ◽  
pp. 559
Author(s):  
Tobial Mchugh ◽  
Karen A. Brown ◽  
Sam J. Daniel ◽  
Sharmila Balram ◽  
Chantal Frigon
Keyword(s):  
Health Care Providers ◽  
Response Rate ◽  
Text Message ◽  
Parental Engagement ◽  
Ambulatory Setting ◽  
Outpatient Basis ◽  
Care Providers ◽  
Electronic Diary ◽  
Pain Diary ◽  
Post Operation

Adenotonsillectomy is performed in children on an outpatient basis, and pain is managed by parents. A pain diary would facilitate pain management in the ambulatory setting. Our objective was to evaluate the parental response rate and the compliance of a prototype electronic pain diary (e-diary) with cloud storage in children aged 2–12 years recovering from adenotonsillectomy and to compare the e-diary with a paper diary (p-diary). Parents recorded pain scores twice daily in a pain diary for 2 weeks post-operation. Parents were given the choice of an e-diary or p-diary with picture message. A total of 208 patients were recruited, of which 35 parents (16.8%) chose the e-diary. Most parents (98%) chose to be contacted by text message. Eighty-one families (47%) returned p-diaries to us by mail. However, the response rate increased to 77% and was similar to that of the e-diary (80%) when we included data texted to the research phone from 53 families. The proportion of diaries with Complete (e-diary:0.37 vs. p-diary:0.4) and Incomplete (e-diary:0.43 vs. p-diary:0.38) data entries were similar. E-diaries provide a means to follow patients in real time after discharge. Our findings suggest that a smartphone-based medical health application coupled with a cloud would meet the needs of families and health care providers alike.

A phase II, multicenter, randomized, open-label study to evaluate the safety and tolerability of proxalutamide (GT0918) in subjects with metastatic castrate-resistant prostate cancer (mCRPC).

2021 ◽  
Vol 39 (6_suppl) ◽  
pp. 108-108
Author(s):  
Tie Zhou ◽  
Dingwei Ye ◽  
Zhongquan Sun ◽  
Qinggui Meng ◽  
Dalin He ◽  
...  
Keyword(s):  
Dose Level ◽  
Response Rate ◽  
Objective Response ◽  
Specific Antigen ◽  
Stage Iv ◽  
Study Drug ◽  
Progression Free Survival ◽  
Open Label ◽  
Significant Difference ◽  
Psa Progression

108 Background: GT0918 is a 2nd generation of AR antagonist and capability to down-regulate AR level. This study is an open-label, randomized, multicenter, Ph II study to evaluate the safety and efficacy in patients with mCRPC, and to determine the optimal dose for Ph III study. Methods: Patients with historically confirmed mCRPC who progressed after/intolerant to/reluctant to receive Docetaxel and previously treated with abiraterone or enzalutamide were excluded. All the patients received up to 6 cycles or, unacceptable toxicity, or loss of clinical benefit as recommended by PCWG3. Primary endpoint was prostate-specific antigen (PSA) reduction from baseline. Secondary endpoints included time to PSA progression (TTPP), objective response rate (ORR), progression free survival (PFS), disease control rate (DCR) and safety profiles. Results: As of June 30, 2019, 108 treated with GT0918 at three dose levels: 100 mg (n =37), 200 mg (n = 35) and 300 mg (n = 36), the median age of patients was 70.0 years (range 63-77), 88% of patients had stage IV disease at the initial diagnosis and 69.4% had Gleason score ≥ 8. The median duration of disease was 2.88 years. All received prior endocrine therapy, 35.2% received prior chemotherapy, 29.6% received Docetaxel. The median PSA at baseline was 35.285ng/ml, PSA response rate (≥50 % reduction from baseline) was 41.9%. The median time to PSA progression was not reached. There was no significant difference among three arms. Of 19 evaluable patients with target lesions at baseline, the ORR was 15.8% (all were PR) assessed by IRC with RECIST v 1.1, with 20.0% (1/5), 22.2% (2/9), 0 (0/5) at 100, 200, 300 dose level, respectively. The DCR assessed by IRC was 78.9% (CR 0+PR15.8%+SD 63.2%). Of 26 evaluable patients with target lesions at baseline, the ORR was 19.2% assessed by investigators (CR 3.8%+PR 15.4%), with 11.1% (1/9), 20.0% (2/10), 28.6% (2/7) at 100, 200, 300 dose level, respectively. Overall, AEs were experienced by most of patients (94.4 %, n=102). AEs leading to drug interruption were reported in 13 patients (12.0%), 9 (8.3%)of them were suspected to be drug related. AEs leading to discontinuation were reported in 6 patients (5.6%), 3(2.8%) were possibly related to GT0918. 14 patients (13.0%) experienced Grade 3 and 4 AEs. 17 patients (15.7%)experienced SAE, 5(4.6%) of them were suspected to be related to study drug. Most of AEs were mild or moderate. The common suspected AE (≥10%) were asthenia (17.6%, n=19), anemia (14.8%, n=16), AST increased (14.8%, n=16), ALT increased (13.0%, n=14), decreased appetite (13.0%, N=14), white blood cell count decreased (12.0%, n=13), proteinuria (12.0%, n=13). Conclusions: GT0918 showed a manageable safety profile. This study provided preliminary anti-tumor activity in patients with mCRPC. 200mg/day is recommended dose for Ph III trial. Clinical trial information: CTR20170177.

scholarly journals Developmental and Behavioral Pediatricians' Attitudes Toward Screening for Fragile X

2013 ◽  
Vol 118 (4) ◽  
pp. 284-293 ◽  
Author(s):  
Kruti Acharya ◽  
Abigail Schindler
Keyword(s):  
Health Care ◽  
Newborn Screening ◽  
Fragile X Syndrome ◽  
Health Care Providers ◽  
Response Rate ◽  
Fragile X ◽  
Educational Interventions ◽  
Care Providers ◽  
Cross Sectional Survey ◽  
Cross Sectional

Abstract Developmental and behavioral pediatricians (DBP) diagnose and care for children with fragile X syndrome. Their attitudes toward FMR1 newborn screening (NBS) and FMR1 carrier testing in childhood could highlight potential pitfalls with FMR1 NBS. We conducted a cross-sectional survey with an adjusted response rate of 61%. Among DBP, 74% supported universal FMR1 NBS, preferring to identify both full mutations and premutations. DBP also support FMR1 testing of asymptomatic siblings. Although DBP support testing for premutations at various points in the lifespan, DBP are not familiar with the array of fragile X–associated disorders (FXAD). Targeted educational interventions are needed to ensure that all health care providers have the knowledge and competence to consent and to counsel families on FXAD.

Effectiveness and Safety of Rizatriptan Benzoate 10 mg in the Treatment of Migraine Headaches

2010 ◽  
Vol 2 ◽  
pp. CMT.S4670
Author(s):  
Michel Aubé ◽  
Fridon Chouha ◽  
Julie Vaillancourt ◽  
John Sampalis
Keyword(s):  
Adverse Events ◽  
Real Life ◽  
Study Drug ◽  
Pain Severity ◽  
Open Label ◽  
Serious Adverse Events ◽  
Over The Counter ◽  
Rizatriptan Benzoate ◽  
Migraine Headaches ◽  
Real Life Setting

Background Patients that do not achieve therapeutic response with over the counter non-triptan medications may benefit from triptan-based treatments. Objective Phase I V, open-label, multi-center, prospective cohort study assessing the effectiveness of rizatriptan in the management of migraines for patients that have not responded to non-triptan treatment. Methods Patients were treated with one rizatriptan (MAXALT RPD®) 10 mg wafer at the onset of each migraine attack and were assessed after a minimum of one and a maximum of two consecutive headache episodes. Outcome measures included self-reported assessments (severity and duration of migraine headache) and the Migraine ACT questionnaire. Results A total of 369 patients were enrolled, of which 291 and 215 reported one and two attacks, respectively. For the first and second attacks, 47.2% and 53.9% of patients reported complete resolution of pain while 73.6% and 77.0% reported pain severity reduction within two hours of onset. Mean (SD) pain severity score (four-point Likert scale) during the 488 migraine episodes was reduced significantly ( P < 0.001) from 2.56 (0.49) at onset to 1.91 (0.85) at 30, 1.31 (1.00) at 60 and 0.84 (1.00) at 120 minutes. Similar improvements were observed for changes in Migraine ACT questionnaire scores. No treatment-related serious adverse events were reported. The most frequently reported non-serious adverse events that were attributed to the study drug were dizziness (2.2%), chest discomfort (1.1%), nausea (1.1%), and somnolence (0.8%). Conclusion In a real-life setting, rizatriptan benzoate 10 mg is effective and safe in the treatment of acute migraine headaches in patients who do not respond to non-triptan treatment.

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