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Case Series of Men with the Germline APC I1307K variant and Treatment-Emergent Neuroendocrine Prostate Cancer

Economides, Minas P; Nakazawa, Mari; Lee, Jonathan W; Li, Xiaochun; Hollifield, Lucas; Chambers, Rachelle; Sarfaty, Michal; Goldberg, Judith D; Antonarakis, Emmanuel S; Wise, David R
INTRODUCTION/BACKGROUND:Somatic mutations in the Wnt signaling gene Adenomatous Polyposis Coli (APC) promote metastatic prostate cancer (PCa) progression. Less is known regarding the impact of germline APC mutations on PCa outcomes. We sought to investigate the prevalence of aggressive variant PCa (AVPC) and treatment-emergent neuroendocrine PCa (t-NEPC) in patients with the germline APC I1307K variant, an alteration found in 7% of Ashkenazi Jewish men. MATERIALS AND METHODS/METHODS:We report a retrospective cohort study comparing patients with PCa and either APC I1307K germline mutation, APC somatic mutations, or unselected patients. Proportions of patients with AVPC among all the cases were estimated along with 95% Clopper-Pearson exact confidence intervals (CI). Odds ratios with 95% CI were provided for the prevalence of t-NEPC and AVPC in patients with germline APC I1307K compared to patients with frameshift alterations in APC. RESULTS:From 2016-2022, 18 patients with PCa at 3 institutions with the germline APC (I1307K) mutation were identified. Clinically-defined AVPC was found in 8 of the 15 cases with metastatic disease (53%; 95% CI: 26%-79%). Combined somatic alterations in two or more of RB1, TP53 or PTEN (molecularly-defined AVPC) were found in 5/18 cases (28%; 95% CI: 10%-54%). When compared to 20 patients with APC somatic frameshift mutations, patients with the germline APC I1307K variant had a significantly increased risk of AVPC (OR 7.2; 95% CI 1.27, 40.68). CONCLUSION/CONCLUSIONS:PCa that develops in the presence of the germline APC I1307K mutation appear to be enriched for clinically-defined and molecularly-defined AVPC and in particular, for t-NEPC.
PMID: 37482523
ISSN: 1938-0682
CID: 5618802

Peer Review of Clinical and Translational Research Manuscripts-Perspectives from Statistical Collaborators

Schulte, Phillip J.; Goldberg, Judith D.; Oster, Robert A.; Ambrosius, Walter T.; Bonner, Lauren Balmert; Cabral, Howard; Carter, Rickey E.; Chen, Ye; Desai, Manisha; Li, Dongmei; Lindsell, Christopher J.; Pomann, Gina Maria; Slade, Emily; Tosteson, Tor D.; Yu, Fang; Spratt, Heidi
Research articles in the clinical and translational science literature commonly use quantitative data to inform evaluation of interventions, learn about the etiology of disease, or develop methods for diagnostic testing or risk prediction of future events. The peer review process must evaluate the methodology used therein, including use of quantitative statistical methods. In this manuscript, we provide guidance for peer reviewers tasked with assessing quantitative methodology, intended to complement guidelines and recommendations that exist for manuscript authors. We describe components of clinical and translational science research manuscripts that require assessment including study design and hypothesis evaluation, sampling and data acquisition, interventions (for studies that include an intervention), measurement of data, statistical analysis methods, presentation of the study results, and interpretation of the study results. For each component, we describe what reviewers should look for and assess; how reviewers should provide helpful comments for fixable errors or omissions; and how reviewers should communicate uncorrectable and irreparable errors. We then discuss the critical concepts of transparency and acceptance/revision guidelines when communicating with responsible journal editors.
SCOPUS:85181827935
ISSN: 2059-8661
CID: 5630072

Peer review of clinical and translational research manuscripts: Perspectives from statistical collaborators

Schulte, Phillip J; Goldberg, Judith D; Oster, Robert A; Ambrosius, Walter T; Bonner, Lauren Balmert; Cabral, Howard; Carter, Rickey E; Chen, Ye; Desai, Manisha; Li, Dongmei; Lindsell, Christopher J; Pomann, Gina-Maria; Slade, Emily; Tosteson, Tor D; Yu, Fang; Spratt, Heidi
Research articles in the clinical and translational science literature commonly use quantitative data to inform evaluation of interventions, learn about the etiology of disease, or develop methods for diagnostic testing or risk prediction of future events. The peer review process must evaluate the methodology used therein, including use of quantitative statistical methods. In this manuscript, we provide guidance for peer reviewers tasked with assessing quantitative methodology, intended to complement guidelines and recommendations that exist for manuscript authors. We describe components of clinical and translational science research manuscripts that require assessment including study design and hypothesis evaluation, sampling and data acquisition, interventions (for studies that include an intervention), measurement of data, statistical analysis methods, presentation of the study results, and interpretation of the study results. For each component, we describe what reviewers should look for and assess; how reviewers should provide helpful comments for fixable errors or omissions; and how reviewers should communicate uncorrectable and irreparable errors. We then discuss the critical concepts of transparency and acceptance/revision guidelines when communicating with responsible journal editors.
PMCID:10879991
PMID: 38384899
ISSN: 2059-8661
CID: 5634432

Factors associated with diagnostic ultrasound for midgut volvulus and relevance of the non-diagnostic examination

El-Ali, Alexander Maad; Ocal, Selin; Hartwell, C Austen; Goldberg, Judith D; Li, Xiaochun; Prestano, Jaimelee; Kamity, Ranjith; Martin, Laura; Strubel, Naomi; Lala, Shailee
BACKGROUND:Few reports explore the frequency and factors associated with diagnostic ultrasound (US) for midgut volvulus. OBJECTIVE:To evaluate predictive factors for diagnostic US for midgut volvulus and clinical outcomes of patients with non-diagnostic US. MATERIALS AND METHODS/METHODS:This retrospective study included infants imaged for midgut volvulus with US. Exams were rated as diagnostic (midgut volvulus present or absent) or non-diagnostic by a pediatric radiologist, and in cases of disagreement with the original report, an additional pediatric radiologist was the tie-breaker. For each exam, the following were recorded: age, weight, respiratory support, exam indication, sonographer experience, and gaseous dilated bowel loops on radiography. Logistic regression models with "stepwise" variable selection were used to investigate the association of diagnostic US for midgut volvulus with each of the independent variables. RESULTS:One hundred nineteen patients were imaged. US was diagnostic in 74% (88/119) of patients. In subsets of patients presenting with bilious emesis or age <28 days, US was diagnostic in 92% (22/24) and 90% (53/59), respectively. Logistic regression suggested that symptom type (bilious vs other) was the best predictor of diagnostic US (type 3 P=0.02). Out of 26 patients with available radiographs, US was diagnostic in 92% (12/13) of patients without bowel dilation on radiographs compared to 62% (8/13) of patients with bowel dilation (P=0.16). Weight, respiratory support, and sonographer experience did not differ between groups. Two sick neonates, ages 2 days and 30 days, in whom the primary clinical concern was dropping hematocrit and sepsis, respectively, had non-diagnostic ultrasounds in the setting of bowel dilation on radiography. Both were found to have midgut volvulus at surgery and both expired. CONCLUSION/CONCLUSIONS:US was most frequently diagnostic in patients with bilious emesis or age less than 28 days. Non-diagnostic US for midgut volvulus must prompt a predetermined follow-up strategy, such as an additional imaging study (e.g., upper GI series), particularly in a sick child, as non-diagnostic US may miss midgut volvulus.
PMID: 37589763
ISSN: 1432-1998
CID: 5619192

PTCy, Abatacept, and Short Course of Tacrolimus for GvHD Prevention Following Haploidentical Transplantation

Al-Homsi, A Samer; Cirrone, Frank; Wo, Stephanie; Cole, Kelli; Suarez-Londono, J Andres; Gardner, Sharon L; Hsu, Jingmei; Stocker, Kelsey; Bruno, Benedetto; Goldberg, Judith D; Levinson, Benjamin A; Abdul-Hay, Maher
Reducing the incidence of graft-versus host disease (GvHD) following haploidentical hematopoietic stem cell transplantation (HSCT) is warranted. Post-transplant cyclophosphamide (PTCy) is the main agent used for GvHD prevention in this setting. It remains unknown if costimulation blockade can be safely combined with PTCy and enhance its efficacy. We performed a phase Ib-II clinical trial to examine the combination of PTCy, abatacept and a short course of tacrolimus (CAST) following peripheral blood haploidentical HSCT. The primary end-point was the incidence of acute GvHD grades II-IV at day +120. The study enrolled 46 patients with a median age of 60 years (range: 18 to 74). The cumulative incidence of acute GvHD grades II-IV and III-IV was 17.4% (95% CI, 9.2% to 32.9%) and 4.4% (95% CI, 1.1% to 17.1%). With a median follow-up of 15.3 months, the cumulative incidence of one-year treatment-related mortality is 4.4% (95% CI, 1.1% to 17.1%). The estimated one-year chronic GvHD moderate to severe rate, relapse rate, progression-free survival, overall survival, and GvHD- and relapse-free survival were 15.9% (95% CI, 8% to 31.7%), 11.7% (95% CI, 5% to 27.2%), 84.1% (95% CI, 73.8% to 95.7%), 85.9% (95% CI, 75.9% to 97.2%) and 66.1% (95% CI, 53.4% to 81.8%), respectively. Toxicities were similar to those expected in patients receiving haploidentical HSCT. This clinical trial showed that CAST regimen is safe and effective in reducing the rate of grades II-IV acute GvHD following haploidentical peripheral blood HSCT (NCT04503616 at https://clinicaltrials.gov/ct2/show/NCT04503616).
PMID: 37163349
ISSN: 2473-9537
CID: 5509352

Crohn's disease phenotype analysis with iodine density from dual-energy CT enterography

Dane, Bari; Li, Xiaochun; Goldberg, Judith D; O'Donnell, Thomas; Le, Linda; Megibow, Alec
PURPOSE/OBJECTIVE:To compare dual-source dual-energy CT enterography (dsDECTE) obtained iodine density (I) (mg/mL) and I normalized to the aorta (I%) with Crohn's disease (CD) phenotypes defined by the SAR-AGA small bowel CD consensus statement. METHODS:Fifty CD patients (31 male, 19 female; mean [SD] age: 50.4 [15.2] years) who underwent dsDECTE were retrospectively identified. Two abdominal radiologists assigned CD phenotypes: no active inflammation (group-2), active inflammation without (group-3) or with luminal narrowing (group-4), stricture with active inflammation (group-5), stricture without active inflammation (group-1), and penetrating disease (group-6). Semiautomatic prototype software was used to determine the median I and I% of CD-affected small bowel mucosa for each patient. The means of the I and I% medians were compared among 4 groups ("1 + 2", "3 + 4", "5", "6") using one-way ANOVA (significance level 0.05 for each outcome) for each outcome individually followed by Tukey's range test for pairwise comparisons with adjusted p-values (overall alpha = 0.05). RESULTS:Mean [SD] I was 2.14 [1.07] mg/mL for groups 1 + 2 (n = 16), 3.54 [1.71] mg/mL for groups 3 + 4 (n = 15), 5.5 [3.27] mg/mL for group- "5" (n = 9), and 3.36 [1.43] mg/mL for group-"6" (n = 10) (ANOVA p = .001; group "1 + 2" versus "5" adj-p = .0005). Mean [SD] I% was 21.2 [6.13]% for groups 1 + 2, 39.47 [9.71]% for groups 3 + 4, 40.98 [11.76]% for group-5, and 35.01 [7.58]% for group-6 (ANOVA p < .0001; groups "1 + 2" versus "3 + 4" adj-p < .0001, group "1 + 2" versus "5" adj-p < .0001, and groups "1 + 2" versus "6" adj-p = .002). CONCLUSION/CONCLUSIONS:Iodine density obtained from dsDECTE significantly differed among CD phenotypes defined by SAR-AGA, with I (mg/mL) increasing with phenotype severity and decreasing for penetrating disease. I and I% can be used to phenotype CD.
PMID: 37097450
ISSN: 2366-0058
CID: 5459552

[S.l.] : Tandem Meetings, Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, 2023

Phase Ib-II Study of Post-Transplant Cyclophosphamide, Abatacept and Short Course of Tacrolimus (CAST) for Graft-Versus-Host Disease Prevention Following Haploidentical Peripheral Blood Stem Cell Transplantation

Al-Homsi, A Samer; Cirrone, Frank; Cole, Kelli; Londono, J Andres-Suarez; Gardner, Sharon; Hsu, Jingmei; Wo, Stephanie; Stocker, Kelsey; Goldberg, Judith; Levinson, Benjamin; Abdul-Hay, Maher
(Website)
CID: 5515802

Phase II study of lenvatinib plus pembrolizumab for patients with immunotherapy-naive advanced gastric cancer following first line therapy [Meeting Abstract]

Cohen, Deirdre Jill; Lee, Jonathan W; Becker, Daniel Jacob; Siolas, Despina; Beri, Nina; Ryan, Theresa; Kozuch, Peter; Yu, Shun; Levinson, Benjamin A; Goldberg, Judith D; Leichman, Lawrence P; Oberstein, Paul Eliezer
ORIGINAL:0016934
ISSN: 1527-7755
CID: 5515782

A Phase II Trial Evaluating Rapid Mid-Treatment Nodal Shrinkage to Select for Adaptive Deescalation in p16+Oropharyngeal Cancer Patients Undergoing Definitive Chemoradiation [Meeting Abstract]

Kim, J. K.; Tam, M.; Karp, J. M.; Oh, C.; Kim, G.; Solomon, E.; Concert, C. M.; Vaezi, A. E.; Li, Z.; Tran, T.; Zan, E.; Corby, P.; Feron-Rigodon, M.; Fitz, C. Del Vecchio; Goldberg, J. D.; Hochman, T.; Givi, B.; Jacobson, A.; Persky, M.; Hu, K. S.
ISI:001079706803134
ISSN: 0360-3016
CID: 5591182

Phase II clinical and immune correlate study of adjuvant nivolumab plus ipilimumab for high-risk resected melanoma

Khushalani, Nikhil I; Vassallo, Melinda; Goldberg, Judith D; Eroglu, Zeynep; Kim, Younchul; Cao, Biwei; Ferguson, Robert; Monson, Kelsey R; Kirchhoff, Tomas; Amato, Carol M; Burke, Paulo; Strange, Ann; Monk, Emily; Gibney, Geoffrey Thomas; Kudchadkar, Ragini; Markowitz, Joseph; Brohl, Andrew S; Pavlick, Anna; Richards, Alison; Woods, David M; Weber, Jeffrey
BACKGROUND:Adjuvant therapy for high-risk resected melanoma with programmed cell-death 1 blockade results in a median relapse-free survival (RFS) of 5 years. The addition of low dose ipilimumab (IPI) to a regimen of adjuvant nivolumab (NIVO) in CheckMate-915 did not result in increased RFS. A pilot phase II adjuvant study of either standard dose or low dose IPI with NIVO was conducted at two centers to evaluate RFS with correlative biomarker studies. METHODS:Patients with resected stages IIIB/IIIC/IV melanoma received either IPI 3 mg/kg and NIVO 1 mg/kg (cohort 4) or IPI 1 mg/kg and NIVO 3 mg/kg (cohorts 5 and 6) induction therapy every 3 weeks for 12 weeks, followed by maintenance NIVO. In an amalgamated subset of patients across cohorts, peripheral T cells at baseline and on-treatment were assessed by flow cytometry and RNA sequencing for exploratory biomarkers. RESULTS:High rates of grade 3-4 adverse events precluded completion of induction therapy in 50%, 35% and 7% of the patients in cohorts 4, 5 and 6, respectively. At a median of 63.9 months of follow-up, 16/56 patients (29%) relapsed. For all patients, at 5 years, RFS was 71% (95% CI: 60 to 84), and overall survival was 94% (95% CI: 88 to 100). Expansion of CD3+CD4+CD38+CD127-GARP- T cells, an on-treatment increase in CD39 expression in CD8+ T cells, and T-cell expression of phosphorylated signal-transducer-and-activator-of-transcription (STAT)2 and STAT5 were associated with relapse. CONCLUSIONS:Adjuvant IPI/NIVO at the induction doses used resulted in promising relapse-free and overall survival, although with a high rate of grade 3-4 adverse events. Biomarker analyses highlight an association of ectoenzyme-expressing T cells and STAT signaling pathways with relapse, warranting future validation. TRIAL REGISTRATION NUMBER:NCT01176474 and NCT02970981.
PMCID:9717375
PMID: 36450385
ISSN: 2051-1426
CID: 5374052