Subcutaneous or intravenous administration of romiplostim in thrombocytopenic patients with myelodysplastic syndrome (MDS)

2009 ◽  
Vol 27 (15_suppl) ◽  
pp. 7009-7009
Author(s):  
M. Sekeres ◽  
H. Kantarjian ◽  
P. Fenaux ◽  
P. Becker ◽  
A. Boruchov ◽  
...  
Keyword(s):  
Treatment Options ◽  
Open Label ◽  
Eligibility Criteria ◽  
Subcutaneous Injections ◽  
Open Label Study ◽  
Future Studies ◽  
Intravenous Injections ◽  
The Past ◽  
The Mean ◽  
Administration Schedules

7009 Background: Thrombocytopenia is common in patients (pts) with MDS, and treatment options are currently limited to platelet (plt) transfusions. Romiplostim is an Fc-peptide fusion protein (peptibody) that stimulates plt production by the same mechanism as thrombopoietin. Methods: This was a phase II, multicenter, single arm, open-label study. Eligibility criteria included IPSS low or intermediate-1 risk MDS and a mean baseline plt count ≤50x109/L. Safety and efficacy of romiplostim were evaluated in pts receiving 750μg romiplostim according to one of 3 schedules: weekly or biweekly subcutaneous injections (QWSC or Q2WSC), or biweekly intravenous injections (Q2WIV). Plt responses were measured per IWG 2006 criteria. Results: Of the 28 pts enrolled, 17 (61%) completed the study; 22 (79%) were male and the mean (±SD) baseline plt count was 29 (±10) x 109/L. The mean age was 71 years and 19 pts (68%) had received plt transfusions in the past year. Mean duration of exposure to romiplostim was 12 (±8) weeks. The most common adverse events (AEs) were fatigue and headache (both 18%). Five pts experienced serious AEs, and there were 2 cases of disease progression to AML: one pt in the QWSC cohort who received romiplostim for 4 weeks and one in the Q2WSC cohort who received romiplostim for 20 weeks. For pts who completed 8 weeks treatment, 15/23 (65%) achieved a plt response, defined by IWG 2006 criteria, and 14/23 (61%) did not require a plt transfusion during this period. The small number of pts limited the ability to compare administration schedules; minor differences were observed in plt responses and plt transfusions, and the incidence of AEs appeared similar among cohorts. Conclusions: IV and SC romiplostim appeared well-tolerated and effective in raising plt counts and avoiding plt transfusions in low and intermediate-1 risk MDS pts. The dose recommendation for future studies is 750μg, given QW or Q2W SC. [Table: see text] [Table: see text]

scholarly journals Maintenance Treatment of Gastroesophageal Reflux Disease: An Evaluation of Continuous and On-Demand Therapy with Rabeprazole 20 mg

2007 ◽  
Vol 21 (12) ◽  
pp. 820-826 ◽  
Author(s):  
David G Morgan ◽  
Michael FJ O’Mahony ◽  
William F O’Mahony ◽  
Jean Roy ◽  
Fernando Camacho ◽  
...  
Keyword(s):  
Gastroesophageal Reflux Disease ◽  
Gastroesophageal Reflux ◽  
Reflux Disease ◽  
Treatment Options ◽  
Open Label ◽  
Open Label Study ◽  
On Demand ◽  
History Of ◽  
The Mean ◽  
Symptom Recurrence

OBJECTIVE: To evaluate continuous therapy (COT) and on-demand therapy (ODT) with rabeprazole 20 mg for maintenance in uninvestigated gastroesophageal reflux disease (GERD).METHODS: This randomized, open-label study enrolled 331 GERD (heartburn-predominant) patients with a pre-existing proton pump inhibitor history of one month or longer, to an acute four-week trial with 20 mg rabeprazole daily for heartburn management. Patients who achieved satisfactory heartburn control during the acute phase (three days or less of heartburn, with no more than one episode rated as moderate, and heartburn rated satisfactorily or completely controlled with minimal rescue antacid use in the seven days preceding randomization) were randomly assigned to six months of rabeprazole 20 mg given as either daily COT or daily ODT, which was initiated upon symptom recurrence and stopped upon symptom resolution. Rescue antacid usage was permitted and tracked. Primary efficacy was measured as the proportion of heartburn-free days over six months.RESULTS: For the 268 patients, the mean percentage of heartburn-free days for the COT group and for the ODT group were 90.3%±14.8% and 64.8%±22.3%, respectively (P<0.0001). COT was associated with an increased number of medication intake days (154±40.2) versus ODT (68±46.1), with less heartburn episodes observed with COT versus ODT, respectively (n=7, n=26, P<0.0001). Ninety-two per cent of COT patients and 79% of ODT patients were either ‘satisfied’ or ‘very satisfied’ with treatment. The mean usage of antacids was low and similar in both groups. COT and ODT regimens were safe and well-tolerated, with a similar incidence of adverse events.CONCLUSION: Results based on symptom assessments favour COT with rabeprazole 20 mg for maintenance therapy in patients with uninvestigated GERD; however, both therapy types are safe and acceptable treatment options for selected patients.

A Preliminary, Open-Label Study of Naltrexone and Bupropion Combination Therapy for Treating Binge Drinking in Human Subjects

2019 ◽  
Author(s):  
T Jordan Walter ◽  
Montserrat Navarro ◽  
Todd E Thiele ◽  
Cort Pedersen ◽  
Alexey Kampov-Polevoy ◽  
...  
Keyword(s):  
Binge Drinking ◽  
Extended Release ◽  
Human Subjects ◽  
Open Label ◽  
Prospective Design ◽  
Open Label Study ◽  
Men And Women ◽  
Average Percentage ◽  
The Past ◽  
Preliminary Study

Abstract Aims The combination of bupropion and naltrexone has shown efficacy in reducing binge drinking in animal models. This study assessed the tolerability and potential utility of combined naltrexone and bupropion in reducing binge drinking in human subjects. Methods This preliminary study employed an open-label, single-arm, 12-week, prospective design. Twelve men and women who exhibited a minimum of five (men) or three (women) binge drinking episodes per month over the past 3 months were recruited. All subjects received both bupropion-extended release 300 mg/day and naltrexone 50 mg/day and were monitored throughout the 3-month treatment period. Binge drinking was assessed using the timeline follow-back method. Results Treatment with combined naltrexone and bupropion reduced the average number of drinks per binge drinking day from 7.8 drinks to 6.4 drinks and reduced the average percentage of binge drinking days per month from 19% (5.7 days/month) to 5% (1.5 days/month). Naltrexone and bupropion were generally well tolerated, with insomnia, headache and nausea/diarrhea being the most common side effects. Six subjects elected to stay on medication after the trial. Conclusions This study suggests that combined naltrexone and bupropion therapy should be further investigated for tolerability and efficacy in reducing binge drinking in humans.

A Multi-Center, Open-Label Study To Evaluate the Safety and Efficacy of Pentostatin, Cytoxan, and Rituxan (PCR) in the Treatment of Previously Untreated or Treated, Stage II, III or IV CLL.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 4978-4978
Author(s):  
Raul R. Mena ◽  
Robert Robles ◽  
Michael Auerbach ◽  
Mehdi M. Moezi ◽  
David Headley
Keyword(s):  
Stage Ii ◽  
Open Label ◽  
Eligibility Criteria ◽  
Bulky Disease ◽  
Old Patients ◽  
Open Label Study ◽  
New Approach ◽  
Study Results ◽  
Intent To Treat ◽  
Anti Cd20

Abstract The decision to treat indolent CLL is often based on progressive bulky disease, worsening symptoms, and increasing hematological derangement. When treatment is indicated, these lymphoproliferative disorders are sensitive to combination immunochemotherapies utilizing pentostatin (P), a purine analog, cyclophosphamide (C), a DNA alkylator, and rituximab (R), an anti-CD20 monoclonal antibody. P+C+R represents a promising new approach in the treatment of patients with CLL. Eligibility criteria allow both treated and treatment-naïve patients diagnosed with Stage II/III/IV CLL (modified Rai classification) to be enrolled. Treatment consisted of intravenous infusions of P (4 mg/m2), C (600 mg/m2), and R (375 mg/m2) on day 1 of a 21-day cycle for a total of up to 10 cycles. Clinical evaluation (including CT scan) was performed after cycles 2, 4, 6, 8 and 10 (if necessary). The intent-to-treat (ITT) population consisted of 70 CLL patients (median age 64, range 35–83) who received a total of 286 cycles (median 4.5 per patient). ECOG status at enrollment was 0 (68%), 1 (28%) and 2 (4%). The overall response rate (CR+Cru+PR) of the 50 evaluable patients was 52% (CR 8%, Cru 14%, PR 30%, SD 46% and PD or RD 2%). 2 grade 4 neutropenia and 1 grade 4 respiratory distress was documented. 4 deaths have been recorded, all in elderly patients (&gt;80 years old), due to acute respiratory failure, myocardial infarction, pulmonary edema and sepsis. This immunochemotherapeutic regimen is active in Stage II/III/IV CLL and the incidence of significant toxicities was low with deaths occurring only in elderly (&gt;80 years old) patients. The study results will be updated at the meeting.

Efficacy of Extracorporeal Photopheresis (ECP) Monotherapy in the Treatment of Cutaneous T Cell Lymphoma.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 2561-2561
Author(s):  
Larisa Geskin ◽  
Francine Foss ◽  
Madeleine Duvic ◽  
David Straus ◽  
Steven Horwitz ◽  
...  
Keyword(s):  
Skin Disease ◽  
Subsequent Treatment ◽  
Topical Steroids ◽  
Open Label ◽  
Eligibility Criteria ◽  
Skin Score ◽  
The Mean ◽  
Hands And Feet

Abstract Background: Mycosis Fungoides (MF) and its leukemic variant Sezary syndrome (SS) are disorders of malignant, skin homing helper/memory T-cells. MF presents with patches, plaques, or tumors, while SS presents with generalized erythroderma and blood involvement. Either can involve lymph nodes, blood, and viscera. A multi-center, open label, single arm clinical trial previously demonstrated the safety and efficacy of ECP as a monotherapy in the treatment of patients with advanced/refractory MF/SS (Edelson, et al, 1987, N Engl J Med, 316:297–303). The primary endpoint of this study was a ≥25% improvement in skin score maintained for at least 4 weeks. We present a long-term, secondary analysis of these patients to further evaluate clinical outcomes and predictors of response for ECP as a monotherapy in MF/SS. Partial (≥50%) and complete (≥90%) skin score responses, extent of skin disease, number of ECP treatments administered, and the time required to achieve 50% and 90% improvement in skin involvement were evaluated. Patients and Methods: Thirty-nine patients (pts) who met eligibility criteria were included in the secondary efficacy analysis as the intent-to-treat (ITT) patient population. Thirty-one pts with generalized erythroderma (GE) and 8 pts with extensive patch plaque (EPP) were treated with ECP on 2 consecutive days every 4–5 weeks for 3 months. No concomitant systemic medications for MF/SS were allowed on study; however, topical steroids could be applied to the hands and feet. Patients had received an average of 3.7 (range 0–13) prior therapies (systemic and topical). Immediately prior to undergoing ECP, all pts received oral doses of methoxsalen in order to achieve blood level concentrations ≥50 ng/mL. Skin improvement was calculated by comparing baseline skin score to skin scores on all subsequent treatment dates. The mean baseline skin score of the 39 ITT patients was 262 (median = 291) based on a maximum possible skin score of 400 points. Results: The median follow-up of the 39 ITT pts was approximately 4 years (range 9 days–7.8 years). Twenty-nine pts (74%) achieved at least a ≥50% improvement in skin score, and 16 pts (41%) achieved ≥90% improvement on ECP monotherapy. The type and extent of skin disease (GE vs. EPP) prior to the start of treatment did not predict response. Patients received a median of 12 (range 4–65) or 30.0 (range 12–109) individual ECP treatments to achieve a ≥50% or ≥90% response, respectively. The mean times to reach a ≥50% or ≥90% response were 8.4 ± 6 months (median=6.5) or 25.2 ±14.9 mos (median=19.6), respectively. The mean duration of a ≥50% response was 32.5 ± 28.6 mos, which included a median of 20 (range 0–153) ECP treatments. Median survival from date of diagnosis and from date of first ECP treatment was 10.6 yrs and 5.4 yrs, respectively. Conclusions: In this long-term, follow-up analysis, ECP monotherapy was associated with a significant and durable improvement in skin score in the majority of patients with MF/SS.

scholarly journals A Randomized, Open-Label Study to Assess Efficacy of Weekly Assumption of Cholecalciferol Versus Calcifediol in Older Patients With Hypovitaminosis D

2021 ◽  
Author(s):  
Chukwuma Okoye ◽  
Valeria Calsolaro ◽  
Filippo Niccolai ◽  
Alessia Maria Calabrese ◽  
Riccardo Franchi ◽  
...  
Keyword(s):  
Vitamin D ◽  
Vitamin D3 ◽  
Older Patients ◽  
Randomized Study ◽  
Hypovitaminosis D ◽  
Clinical History ◽  
Open Label ◽  
Open Label Study ◽  
The Mean

The aim of this single-center, open-label, non-controlled randomized study was to evaluate which formulation of vitamin D between cholecalciferol and calcifediol is most effective in the treatment of hypovitaminosis D in older adults. Demographic characteristics, clinical history and comprehensive geriatric assessment were recorded at admission. Eligible patients randomly received an equivalent vitamin D supplement either with cholecalciferol or calcifediol from hospital admission to three months after discharge. Among the 140 older patients included (mean age 83&plusmn;6.6, 57.8% females), 69 received cholecalciferol and 71 calcifediol. The mean plasma values of 25OH-Vitamin D3 found at the enrollment were 16.8 &plusmn; 9.9 ng/mL in patients receiving cholecalciferol and 18.8 &plusmn; 13.3 ng/mL in those treated with calcifediol (p =0.31). At the 3-month follow up, the mean concentration of 25OH-Vitamin D3 was significantly higher in patients treated with calcifediol than in patients treated with cholecalciferol (respectively, 30.7 &plusmn; 8.4 vs 45.4 &plusmn; 9.8 ng/mL, p &amp;lt;0.001). Supplementation with cholecalciferol or calcifediol results in both cases effective in reaching optimal circulating values of 25OH-VitaminD3 in the older patients suffering from hypovitaminosis D. However, supplementation with calcifediol led to average circulating values of 25OH- VitaminD3 significantly higher (over 50%) than those obtained with cholecalciferol.

Evidence-Based Treatments

2017 ◽  
Author(s):  
W. Curt LaFrance ◽  
Laura H. Goldstein
Keyword(s):  
Treatment Options ◽  
Controlled Trials ◽  
Limited Sample ◽  
Future Studies ◽  
Psychogenic Nonepileptic Seizures ◽  
The Past ◽  
Evidence Based Treatments ◽  
Nonepileptic Seizures ◽  
New Treatment ◽  
Functional Neurological Disorders

Psychogenic nonepileptic seizures (PNES) have been in the medical literature for centuries. However, treatments were limited, being based on uncontrolled data, until the past decade. Treatment advances published since 2010 have included pilot controlled trials using psychotherapies, psychoeducational approaches, medications, and combined pharmacological and psychotherapeutic approaches that provide new treatment options for patients with PNES. This chapter describes these controlled trials in detail. It also covers studies of treatments for other functional neurological disorders including PNES. One conclusion from this review is that future studies still need to improve on as-yet limited sample sizes and provide insights into predictors of treatment outcome so that rational decisions can be made about which treatments offer the best outcome and who is likely to best respond to which treatment.

scholarly journals Pharmacologic Treatment for Pediatric Gastroparesis: A Review of the Literature

2016 ◽  
Vol 21 (2) ◽  
pp. 120-132 ◽  
Author(s):  
Emma M. Tillman ◽  
Keaton S. Smetana ◽  
Likeselam Bantu ◽  
Merrion G. Buckley
Keyword(s):  
Pharmacological Treatment ◽  
Treatment Options ◽  
Disease State ◽  
Adult Patients ◽  
Clear Correlation ◽  
Pharmacologic Treatment ◽  
Review Of The Literature ◽  
Future Studies ◽  
The Past ◽  
Associated Symptoms

There have been a number of agents that have been tried for treatment of gastroparesis over the past 3 decades, with varying levels of success. Guidelines exist for the management of gastroparesis in adults; however, even though the cause of gastroparesis in children is similar to that in adults, no guidelines exist for treating pediatric gastroparesis as studies on the topic are limited. With what little information we have on pediatric gastroparesis, medications used in children's studies do not seem to demonstrate the same results as in adult patients with gastroparesis; thus, future studies of whether certain medications are effective for treating pediatric gastroparesis and at what dose still need to be conducted. Pharmacological treatment options for pediatric gastroparesis do not show a clear correlation of resolving or even maintaining gastroparesis-associated symptoms or disease state. This article reviews the available studies of drugs that have shown some efficacy, with an emphasis on pediatric studies.

Azacitidine in the Treatment of Elderly Patients with Acute Myelogenous Leukemia

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 4041-4041 ◽  
Author(s):  
George Labban ◽  
James M. Rossetti ◽  
Richard K. Shadduck ◽  
Entezam Sahovic ◽  
Haifaa Abdulhaq ◽  
...  
Keyword(s):  
Elderly Patients ◽  
Acute Myelogenous Leukemia ◽  
Myelogenous Leukemia ◽  
Open Label ◽  
Open Label Study ◽  
Acute Myelogenous ◽  
Prospective Phase ◽  
Blast Count ◽  
The Mean ◽  
To Receive

Abstract BACKGROUND: Effective treatment of the elderly patient with acute myelogenous leukemia (AML) remains a challenging task. Elderly patients with AML usually respond poorly to standard induction chemotherapy. Response rates in elderly patients are in the range of 30–50% compared to 80–90% in younger patients. Moreover, prolonged hospitalization with treatment related mortality as high as 30% is typical in this older population. In a prior retrospective analysis done at our institution, azacitidine showed an overall response rate of 60% with limited toxicity when administered to patients older than 55 years of age with AML. We present an interim analysis of the first 8 patients enrolled in our prospective, phase II open label study using single agent azacitidine for elderly patients with AML. METHODS: This is a prospective, phase II open label study using azacitidine in patients ≥ 60 years with AML. Inclusion criteria: Newly diagnosed AML (de novo or secondary, WHO criteria) and ECOG ≤ 2. Promyelocytic (M3) phenotype was excluded. Patients with circulating blast count ≥ 30,000/mcl were treated with hydroxyurea until &lt; 30,000/mcl. Azacitidine was given at a dose of 100 mg/m2 subcutaneously for 5 consecutive days every 28 days until disease progression or significant toxicity. G-CSF was given to patients with neutropenia (ANC &lt; 1000/mcl) during all cycles excluding cycle one. RESULTS: Eight patients have been enrolled to date. The mean age of patients is 74 (range: 64–82 years). The mean baseline ECOG performance score was 1 with a mean during treatment of 1. Mean baseline bone marrow blast count was 53% (range: 21–92%). Overall response rate using the NCI response criteria was 75% (6/8): complete response (CR; n=2; 25%) and partial response (PR; n=4; 50%). The mean number of days on treatment was 117 (range: 4–247 days). The mean number of days hospitalized during therapy was 18 (range: 7–51 days) with the majority of therapy being given in the outpatient setting. The mean overall survival time from diagnosis for all patients was 180 days (range: 23–403). The mean overall survival time for responders was 200 days (range: 36–403). Three patients continue on therapy at 146 (PR), 153 (CR) and 247 (PR) days. Of the other responders, one went on to receive an allogeneic PBSCT, one died at 36 days from complications of a strangulated hernia, and one removed himself from study at 82 days (unconfirmed CR) to receive treatment closer to home. All patients were red blood cell (RBC) transfusion dependent at the start of the therapy. To date, two of the six responders (33%) became independent of RBC transfusion. Four patients were transfusion dependent for platelets at the start of therapy with two being non-responders and two achieving a PR. Non-hematological toxicity was limited to mild injection site skin reaction and fatigue in 63% (5/8) each. No treatment related deaths were observed. The dose and schedule of therapy remained constant in all patients except one who required a 25% dose reduction after cycle 3 due to drug induced marrow suppression. CONCLUSION: This interim analysis suggests that the administration of subcutaneous azacitidine in an accelerated dosing schedule to elderly patients with acute myelogenous leukemia is a feasible and well-tolerated alternative to standard induction chemotherapy.

scholarly journals 1393. A Phase 1, Randomized, Open-Label, Crossover Study in Healthy Subjects Under Fasting Conditions of Orally Administered Sulopenem Etzadroxil Alone or with Probenecid to Determine the Pharmacokinetics of Sulopenem

2018 ◽  
Vol 5 (suppl_1) ◽  
pp. S428-S429
Author(s):  
Michael Dunne ◽  
Steven Aronin ◽  
Elise Dunzo ◽  
Sailaja Puttagunta
Keyword(s):  
Oral Dose ◽  
Single Oral Dose ◽  
Phase 1 ◽  
Treatment Options ◽  
Parent Compound ◽  
Additional Treatment ◽  
Open Label ◽  
Males And Females ◽  
The Mean ◽  
Mean Time

Abstract Background Antimicrobial resistance to available oral antibiotics is becoming progressively more common, precipitating the need for additional treatment options as step-down from initial intravenous (IV) therapy as well as for treatment of infections in the community. Sulopenem (CP-70,429) is a thiopenem antibiotic active against quinolone non-susceptible and ESBL-producing Enterobacteriaceae. As the key pharmacokinetic-pharmacodynamic variable correlating with efficacy for penem antibiotics is time above minimum inhibitory concentration (T &gt; MIC), we examined the utility of probenecid, an OAT-1 inhibitor of β-lactam excretion, on the pharmacokinetic (PK) parameters for the oral prodrug of sulopenem, sulopenem etzadroxil Methods Twelve healthy males and females received a single oral dose of 500 mg sulopenem etzadroxil as powder in bottle either alone or co-administered with a single oral dose of probenecid 500 mg in a crossover design with a washout period of 6 days. All doses were administered under fasting conditions. Blood samples for plasma PK analysis were collected and PK parameters for sulopenem, the parent compound of sulopenem etzadroxil, were determined. Results Treatment Conclusion Probenecid increases the AUC of sulopenem by 28% in the fasted state and extends the mean time over MIC. Disclosures M. Dunne, Iterum Therapeutics: Employee and Shareholder, Salary. S. Aronin, Iterum Therapeutics: Employee and Shareholder, Salary. E. Dunzo, Parexel: Consultant, Consulting fee. S. Puttagunta, Iterum Therapeutics: Employee and Shareholder, Salary.

The Effect of Fenoprofen Calcium (Nalfon) on Levels of Rheumatoid Factor in Patients with Rheumatoid Arthritis

1973 ◽  
Vol 1 (2) ◽  
pp. 412-416
Author(s):  
Joan E Himes ◽  
Ivan F Duff
Keyword(s):  
Rheumatoid Arthritis ◽  
Rheumatoid Factor ◽  
Open Label ◽  
Open Label Study ◽  
Label Study ◽  
The Mean ◽  
Baseline Levels ◽  
One Year

In 88.9% of a group of 18 patients with rheumatoid arthritis participating in an open-label study with fenoprofen calcium (Nalfon), there was observed a mean litre decrease in level of rheumatoid factor as compared to baseline levels. In follow-up, three months to one year later, a uniform litre increase above the mean level of the study period had occurred. In all cases the titre returned to baseline levels or higher.

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