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Boca Raton, FL, United States

Gregory S.A.,Rush University | Schwartzberg L.S.,The West Clinic | Mo M.,Amgen Inc. | Sierra J.,Hospital de la Santa Creu i Sant Pau | Vogel C.,Boca Research
Community Oncology | Year: 2010

Bone pain associated with pegfilgrastim has been perceived to occur at higher frequency and with greater severity than bone pain associated with filgrastim. This retrospective study analyzed patient-level data from randomized controlled trials (RCTs) conducted by Amgen to compare bone pain incidence in patients receiving: (1) once-per-chemotherapy-cycle pegfilgrastim (100 μg/kg or 6-mg fixed dose) versus daily filgrastim (5 μg/kg) in seven studies, and (2) once-per-chemotherapy- cycle pegfilgrastim (100 μg/kg or 6 mg) versus no granulocyte colony-stimulating factor (G-CSF) in five studies. An exploratory analysis that included patients who received once-per-chemotherapy-cycle pegfilgrastim (100 μg/kg or 6 mg) in 23 RCTs, non-RCTs, or single-arm interventional trials was also performed. In RCTs that compared pegfilgrastim versus filgrastim, the incidence (95% CI) of any grade and grade 3/4 bone pain was similar in the pegfilgrastim and filgrastim groups for the first four chemotherapy cycles: any grade, 62% (57%, 67%) and 66% (61%, 71%); grade 3/4, 7% (4%, 10%) and 8% (5%, 11%), respectively. In RCTs that compared pegfilgrastim versus no G-CSF, patients receiving pegfilgrastim appeared to have experienced more bone pain of any grade in the first chemotherapy cycle than patients not receiving G-CSF: 33% (30%, 36%) versus 23% (21%, 26%). Grade 3/4 bone pain was infrequently reported in the first cycle for both treatment arms: 3% (2%, 5%) versus 2% (1%, 3%). Results from subgroup analyses suggest that bone pain may be associated with age, tumor type, and chemotherapy regimen. In this retrospective analysis, bone pain associated with pegfilgrastim did not occur at higher frequency or with greater severity than bone pain associated with filgrastim. © 2010 Elsevier Inc. Source


Weinreb N.J.,Boca Research | Weinreb N.J.,University of Miami | Lee R.E.,University of Pittsburgh
Critical Reviews in Oncogenesis | Year: 2013

Patients with type 1 Gaucher disease (GD1) have increased risk of developing myeloma, other hematological cancers, hepatocellular carcinoma, and other solid tumors. Patient awareness of the GD1-cancer association causes anxiety and fear. Little is known about cancer as a cause of death in GD1, especially in patients never treated with GD1-specific therapies. Consequently, the effect of treatment on cancer mortality in GD1 patients is difficult to evaluate. In this review, starting with a population of 184 GD1 cases never treated, we annotate and analyze the causes of death of 57 GD1 patients who died of cancer. The proportional mortality ratio (PMR) for all malignancies in patients with GD1 is 1.57 (p=0.0002), but it is much higher for myeloma (PMR = 9.66) and other hematological cancers, hepatocellular carcinoma, and kidney cancer (PMR = ~4). However, deaths from colorectal and pancreatic cancers were not more frequent than expected, and deaths from lung, breast, gynecological, and prostate cancer occurred less than anticipated. Herein, we discuss whether GD1 is truly a hereditary cancer syndrome and the problem of comorbidities and cancer risk assessment, and we speculate as to whether the variability in death by cancer type might be attributable to biochemical sequelae of tumor cell and macrophage/stromal cell GBA1 mutation affecting signals for metastasis, the process most closely associated with cancer mortality. © 2013 by Begell House, Inc. Source


Weinreb N.J.,Boca Research | Finegold D.N.,Childrens Hospital of Pittsburgh | Feingold E.,University of Pittsburgh | Zeng Z.,University of Pittsburgh | And 3 more authors.
Orphanet Journal of Rare Diseases | Year: 2015

Background: GD1-DS3 is an integrated assessment of type 1 Gaucher disease (GD1) burden based on bone, hematologic and visceral domains. We investigated this disease severity scoring system (DS3) methodology for initial assessment, long-term follow-up and evaluation of treatment responses. Methods: We enrolled 133 treated adult GD1 patients. Baseline DS3 scores were calculated near the initial treatment date and patients stratified by severity as marked (DS3 6.00-19.00), moderate (DS3 3.00-5.99), mild (DS3 < 3.00). Follow-up scores were calculated annually. Minimal clinically important improvement (MCII), is defined as ΔDS3 of -3.1. Results: Patient characteristics: N370S was the most common allele (118 patients had at least one), 52 were N370S/N370S (48/52 were Ashkenazi Jews), N370S/L444P was the most common genotype among non-Jews. Median age of treatment: 45 years; median follow-up: 14 years. Baseline DS3 scores: Patients with marked disease (N = 58; median 7.84) were least likely to be N370S homozygous (19 %) and most likely to have had splenectomy (53 %), early age at diagnosis (median 18 years) and major pre-treatment bone pathology (76 %). Among patients with moderate disease (N = 53; median 4.33), 49 % were N370S/N370S, 15.1 % had splenectomy and 17 % had major bone disease. Median age at diagnosis: 32 years. No patient with mild disease (N = 22; median 2.4) had splenectomy or major skeletal disease. Median age at diagnosis: 40 years. 68 % were N370S homozygous. Response to treatment: Health-state transitions occurred primarily during the early treatment years. At Year 5, among 48 evaluable patients with marked baseline disease, eight were unchanged in severity status whereas 40 had MCII of varying degrees with 11 scored as mild. Among 42 evaluable moderate patients, none worsened, 16 remained moderate and 26 improved to mild. Among 16 evaluable mild patients, 14 remained so and 2 had DS3 scores in the low moderate range. Conclusions: DS3 is effective for assessing disease burden in GD1 and for monitoring response. ERT was associated with MCII in DS3 scores in patients with high severity. Nevertheless, despite better DS3 scores with treatment, GD1 patients especially those with splenectomy and pre-treatment bone pathology, continued to have bone complications. © 2015 Weinreb et al. Source


Weinreb N.J.,Boca Research | Finegold D.N.,One Childrens Hospital Drive | Feingold E.,University of Pittsburgh | Zeng Z.,University of Pittsburgh | And 3 more authors.
Orphanet Journal of Rare Diseases | Year: 2015

Background: GD1-DS3 is an integrated assessment of type 1 Gaucher disease (GD1) burden based on bone, hematologic and visceral domains. We investigated this disease severity scoring system (DS3) methodology for initial assessment, long-term follow-up and evaluation of treatment responses. Methods: We enrolled 133 treated adult GD1 patients. Baseline DS3 scores were calculated near the initial treatment date and patients stratified by severity as marked (DS3 6.00-19.00), moderate (DS3 3.00-5.99), mild (DS3 < 3.00). Follow-up scores were calculated annually. Minimal clinically important improvement (MCII), is defined as {increment}DS3 of -3.1. Results: Patient characteristics: N370S was the most common allele (118 patients had at least one), 52 were N370S/N370S (48/52 were Ashkenazi Jews), N370S/L444P was the most common genotype among non-Jews. Median age of treatment: 45 years; median follow-up: 14 years. Baseline DS3 scores: Patients with marked disease (N = 58; median 7.84) were least likely to be N370S homozygous (19 %) and most likely to have had splenectomy (53 %), early age at diagnosis (median 18 years) and major pre-treatment bone pathology (76 %). Among patients with moderate disease (N = 53; median 4.33), 49 % were N370S/N370S, 15.1 % had splenectomy and 17 % had major bone disease. Median age at diagnosis: 32 years. No patient with mild disease (N = 22; median 2.4) had splenectomy or major skeletal disease. Median age at diagnosis: 40 years. 68 % were N370S homozygous. Response to treatment: Health-state transitions occurred primarily during the early treatment years. At Year 5, among 48 evaluable patients with marked baseline disease, eight were unchanged in severity status whereas 40 had MCII of varying degrees with 11 scored as mild. Among 42 evaluable moderate patients, none worsened, 16 remained moderate and 26 improved to mild. Among 16 evaluable mild patients, 14 remained so and 2 had DS3 scores in the low moderate range. Conclusions: DS3 is effective for assessing disease burden in GD1 and for monitoring response. ERT was associated with MCII in DS3 scores in patients with high severity. Nevertheless, despite better DS3 scores with treatment, GD1 patients especially those with splenectomy and pre-treatment bone pathology, continued to have bone complications. © 2015 Weinreb et al. Source


Rendon M.I.,Boca Research | Rendon M.I.,University of Miami
Journal of Cosmetic Dermatology | Year: 2012

Background Facial rejuvenation techniques have evolved in recent decades driven by a paradigm shift to restoration of lost volume, and an increase in the number of available products. As clinical experience has increased, practitioners have further refined the use of these products. Objectives To share observations and practical recommendations based on clinical experience with the aesthetic use of injectable poly-l-lactic acid (PLLA) in patients followed for up to 5years. Methods Literature review and retrospective case history review of the first 100 patients treated with injectable PLLA, many of whom have been followed for over 5years. Results Use of injectable PLLA, alone or in combination with other products, has provided excellent and long-lasting (up to 5years) aesthetic results for most of the reviewed patients. Typically, patients have received one or two touch-up sessions between years 2 and 4 after initial full correction. There were minimal adverse events of papule formation; these eventually resolved. Conclusions Injectable PLLA is a safe and effective option for achieving long-term patient satisfaction with soft-tissue augmentation. © 2012 Wiley Periodicals, Inc. Source

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