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279 citation(s) found for "Stephens, DH".

Gastrointestinal motility evaluation in children with orthostatic intolerance: Mayo Clinic experience
Manini, Mhd Louai , Barazi, Adnan, Khemani, Disha, Abd‐Rabu, Rami , Absah, Imad , Mara, Kristin C. , Stephens, Michael C., Camilleri, Michael , Fischer, Philip R. , Mara, Kristin, Stephens, Michael, Camilleri, M , Mara, Kristin C

Mayo Clinic

Neurogastroenterology & Motility | June 2, 2020 | Volume: 32 | Issue: 8 | Pages: No page available

10.1111/nmo.13863 | PMID 32483845 Get It

AbstractObjectiveOrthostatic intolerance (OI) and autonomic dysfunction (AD) are common in adolescents and young adults. Patients experience multisystem symptoms including gastrointestinal (GI), postural orthostatic tachycardia syndrome (POTS), orthostatic hypotension (OH), or only symptoms of OI (SOI) without significant findings on 70‐degree head‐up tilt testing (HUT). We hypothesize that patients with POTS, OH, and SOI show differences in GI symptoms and motility test and that heart rate (HR) changes on HUT predict severity of GI dysmotility.Study designFrom medical records of patients (<18 years) with OI, we collected demographics, presenting symptoms, GI manifestations, and GI motility testing. Data were compared between the 3 groups (POTS, OH, and SOI). We assessed changes in HR on HUT with changes on GI motility evaluation.ResultsTwo hundred twenty‐nine patients were included (73% females). Abdominal pain (65%), nausea (49%), vomiting (18%), and constipation (24%) were the most common GI symptoms. In patients who had motility evaluation, there were 27% (53/193) with delayed gastric emptying (GE) at 4 hours, 35% (32/92) with delayed colonic transit (CT), 55% (17/31) with reduced gastric accommodation (GA), and 75% (21/28) with dyssynergic defecation (DD). Among 100 POTS, 34 OH, and 95 SOI patients, no significant differences in GI symptoms or motility tests were identified and HR changes on HUT were not associated with changes on motility tests.ConclusionGI symptoms are frequent in adolescents with OI and are associated with delayed GE, reduced GA, delayed CT, and presence of DD.
Natural History of  Very Early Onset Inflammatory Bowel Disease in North America: A Retrospective Cohort Study
Kerur, Basavaraj , Benchimol, Eric I. , Fiedler, Karoline , Stahl, Marisa G. , Hyams, Jeffrey S. , Stephens, Michael C., Lu, Ying , Pfefferkorn, Marian, Alkhouri, Raza, Strople, Jennifer , Kelsen, Judith R. , Siebold, Leah, Goyal, Alka , Rosh, Joel R. , LeLeiko, Neal S. , Limbergen, Johan Van , Guerrerio, Anthony L. , Maltz, Ross , Karam, Lina , Crowley, Eileen , Heyman, Melvin B. , Benkov, Keith , Noe, Joshua D. , Mouton, Dedrick, Pappa, Helen, Galanko, Joseph A. , Muise, Aleixo M. , Stephens, Michael, Leleiko, Neal S , Griffiths, Anne Marie , Deneau, Mark , Snapper, Scott B. , Kappelman, Michael D.

Mayo Clinic

Inflammatory Bowel Diseases | May 9, 2020 | Volume: 27 | Issue: 3 | Pages: 295-302

10.1093/ibd/izaa080 | PMID 32386060 Get It

Abstract Background The incidence of very early onset inflammatory bowel disease (VEOIBD) is increasing, yet the phenotype and natural history of VEOIBD are not well described. Methods We performed a retrospective cohort study of patients diagnosed with VEOIBD (6 years of age and younger) between 2008 and 2013 at 25 North American centers. Eligible patients at each center were randomly selected for chart review. We abstracted data at diagnosis and at 1, 3, and 5 years after diagnosis. We compared the clinical features and outcomes with VEOIBD diagnosed younger than 3 years of age with children diagnosed with VEOIBD at age 3 to 6 years. Results The study population included 269 children (105 [39%] Crohn’s disease, 106 [39%] ulcerative colitis, and 58 [22%] IBD unclassified). The median age of diagnosis was 4.2 years (interquartile range 2.9–5.2). Most (94%) Crohn’s disease patients had inflammatory disease behavior (B1). Isolated colitis (L2) was the most common disease location (70% of children diagnosed younger than 3 years vs 43% of children diagnosed 3 years and older; P = 0.10). By the end of follow-up, stricturing/penetrating occurred in 7 (6.6%) children. The risk of any bowel surgery in Crohn’s disease was 3% by 1 year, 12% by 3 years, and 15% by 5 years and did not differ by age at diagnosis. Most ulcerative colitis patients had pancolitis (57% of children diagnosed younger than 3 years vs 45% of children diagnosed 3 years and older; P = 0.18). The risk of colectomy in ulcerative colitis/IBD unclassified was 0% by 1 year, 3% by 3 years, and 14% by 5 years and did not differ by age of diagnosis. Conclusions Very early onset inflammatory bowel disease has a distinct phenotype with predominantly colonic involvement and infrequent stricturing/penetrating disease. The cumulative risk of bowel surgery in children with VEOIBD was approximately 14%–15% by 5 years. These data can be used to provide anticipatory guidance in this emerging patient population.
Meeting the Educational Needs of an Increasingly Diverse Surgical Workforce
Stephens, Elizabeth H. , Blackmon, Shanda H, Antonoff, Mara B. , Blackmon, Shanda

Mayo Clinic

JAMA Surgery | March 18, 2020 | Volume: 155 | Issue: 6 | Pages: 533

10.1001/jamasurg.2020.0097 | PMID 32186686 Get It

Prevalence and Predictors of Climacturia and Associated Patient/Partner Bother in Patients with History of Definitive Therapy for Prostate Cancer
Jimbo, Masaya , Alom, Manaf, Pfeifer, Zachary, Haile, Eiftu , Stephens, Dane, Gopalakrishna, Ajay , Ziegelmann, Matthew , Trost, Landon , Köhler, Tobias , Ziegelmann, Matthew J, Viers, Boyd R. , Trost, Landon W

Mayo Clinic

The Journal of Sexual Medicine | March 13, 2020 | Volume: 17 | Issue: 6 | Pages: 1126-1132

10.1016/j.jsxm.2020.02.016 | PMID 32179016 Get It

Abstract Background Climacturia is an under-reported complication of definitive therapy for prostate cancer (PCa) - that is, radical prostatectomy (RP) and/or radiation therapy (RT). Aim We sought to identify the prevalence and predictors of climacturia and associated patient/partner bother in patients with and without prior PCa treatment. Methods We analyzed a database of patients who presented to our Men's Health clinic and filled out a questionnaire related to sexual function and pertinent medical histories. The prevalence of climacturia and associated patient/partner bother in patients with/without prior RP/RT was calculated. Univariable and multivariable logistic regressions were performed to identify predictors associated with climacturia and patient/partner bother. Outcomes The primary outcomes were the prevalence and predictors of climacturia and associated patient/partner bother in patients with/without history of definitive PCa treatment. Results Among 1,117 patients able to achieve orgasm, 192 patients (17%) had prior history of definitive therapy for PCa (RP alone = 139 [72%]; RT alone = 22 [11%]; RP + RT = 31 [16%]). Climacturia was reported by 39%, 14%, 52%, and 2.4% of patients with history of RP alone, RT alone, RP + RT, and neither RP nor RT, respectively (P &lt; .05 between all groups). 33 to 45 percent of patients with climacturia noted significant patient/partner bother. Factors significantly associated with climacturia were prior RP, prior RT, history of other prostate surgery, and erectile dysfunction, although erectile dysfunction was not significant on multivariable analysis. Significant reduction in climacturia prevalence was noted for patients who were ≥1 year out from RP, compared with patients who were &lt;1 year out. Among patients with prior RP/RT, stress urinary incontinence was associated with increased risk of climacturia, whereas diabetes was associated with decreased risk. No factors were associated with patient/partner bother. Among patients with prior RP, nerve-sparing technique did not predict presence of climacturia but was associated with reduced patient/partner bother. Clinical translation Given significant prevalence of climacturia and associated patient/partner bother, patients should be counseled on the risk of climacturia before undergoing RP/RT. Strengths and limitations Strengths include the large study population and the focus on both RP and RT. Limitations include the facts that this is a single-institution study that primarily relies on patients’ subjective reporting and that the study population may not represent the general population. Conclusions Climacturia affects a significant proportion of patients with history of RP/RT for PCa, and many patients and their partners find this bothersome.
PET-Directed Therapy for Patients with Limited-Stage Diffuse Large B-Cell Lymphoma - Results of Intergroup Nctn Study S1001
Persky, Daniel O. , Swinnen, Lode J., Winegarden, Jerome D., Leonard, John P. , Song, Joo Y. , Fisher, Richard I., Smith, Sonali M. , Friedberg, Jonathan W. , Song, Joo Y , Li, Hongli , Stephens, Deborah M. , Park, Steven , Bartlett, Nancy L. , Barr, Paul M. , Constine, Louis S , FitzGerald, Thomas J. , Kahl, Brad S. , LeBlanc, Michael , Rimsza, Lisa M.

Mayo Clinic

Blood | February 14, 2020 | Volume: 134 | Issue: Supplement_1 | Pages: 349-349

10.1182/blood-2019-123871 Get It

Introduction Limited stage disease accounts for 30-40% of Diffuse Large B-cell Lymphoma (DLBCL), with better overall survival than advanced stage disease, but with increased late relapses regardless of treatment strategy (Stephens 2016). Preferred treatment for these patients (pts) per NCCN guidelines is abbreviated R-CHOP followed by radiotherapy. Based on promising results of radioimmunotherapy consolidation in SWOG S0313 (Persky 2015), and of PET-directed experience by the BC Cancer Agency (Sehn 2011), we designed a National Clinical Trials Network (NCTN) PET-directed study to tailor therapy after 3 cycles of R-CHOP, to improve outcomes and decrease toxicities. Methods Pts had non-bulky (&lt; 10 cm) stage I/II untreated DLBCL. Mediastinal, HIV-associated, testicular, central nervous system, and indolent lymphoma were excluded. Pts received standard R-CHOP therapy and had an interim PET scan on day 15-18 of cycle 3, which was centrally reviewed in real time. Pts with negative PET scan (Deauville 1-3, iPET-neg) proceeded with 1 additional cycle of R-CHOP. Pts with positive PET scan (Deauville 4-5, iPET-pos) initiated 36 Gy of involved field radiation therapy (IFRT), plus additional boost to FDG-avid area up to 9 Gy, within 5 weeks of 3rd cycle of R-CHOP. This was followed by ibritumomab tiuxetan (IFRT-Zevalin) 3-6 weeks after completing IFRT. Final PET scan was performed 12 weeks after treatment completion. Results The study completed accrual in June 2016. Safety, response, interim PET, and immunohistochemistry-based cell of origin (COO) and MYC/BCL2 analyses were presented previously (Persky 2017, Stephens 2017). Of 159 pts enrolled, 1 was upstaged by PET, and 26 pts were ineligible - due to incorrect histology (mostly concurrent indolent or follicular lymphoma grade 3B) (21), lack of diagnostic tissue submission for central pathology review (3), and bulky disease (2). In 132 eligible pts, median age was 62 years, 62% had stage I, 17% had B symptoms, 14% had elevated LDH, 43% had extranodal involvement, and 66% had exclusive involvement of the head and neck region. Stage modified IPI (smIPI, Miller 1998) was 0 in 27%, 1 in 42%, 2 in 28%, and 3 in 4% of the pts. COO by Lymph2Cx was assessable in 87 pts - 68% were GCB, 23% were ABC, and 9% were unclassifiable. Double protein expressers (MYC and BCL2, DPE) were 16%, while 4 (3%) pts had "double hit" lymphoma (DHL) - 2 with MYC/BCL2 and 2 with MYC/BCL6 rearrangements - none of which were DPE. Of 132 eligible pts, 128 had an interim PET scan centrally reviewed, of which 110 were iPET-neg. Only 18 were iPET-pos, 4 of them due to infection (Deauville X) which were treated as iPET-neg. Of 14 truly iPET-pos pts (11%), 2 refused radiation, and 12 pts received IFRT-Zevalin. Eight pts (67%) converted from PR to CR after IFRT-Zevalin and 4 (33%) had PR, for an overall CR of 92% and PR of 4% (with 4% unevaluable). With median follow up of 4.5 yrs (range 1.1 - 7.5 yrs), only 5 pts progressed and 2 died from lymphoma. Of 5 progressors, 3 received R-CHOP x 4, 1 was iPET-pos but declined radiation, and 1 went off treatment after 1 cycle of R-CHOP. Eleven pts died from non-lymphoma causes, including 1 pt from secondary AML (iPET-neg arm), and 1 of lung adenocarcinoma diagnosed upon iPET. S1001 5-yr PFS estimate is 87% and OS estimate is 90% (figure 1), with iPET-pos and iPET-neg pts having similar outcomes - PFS 86% vs. 88%, OS 93% vs. 91%, respectively. Five-yr PFS by smIPI was 97% for smIPI of 0, 86% for 1-2, and 30% for 3. GCB had 5-yr PFS of 95%, vs. 70% for ABC and 47% for unclassifiable. DPE pts had 5-yr PFS of 70, vs. 89% for non-DPE pts. All 4 DHL pts maintain remission. Conclusions S1001 is the largest US study of limited stage DLBCL in the rituximab era, with best NCTN results in this disease subset. Only 5 patients progressed and 2 died from lymphoma. Our study confirms the distinct biology of limited stage DLBCL, with predominance of GCB origin (68%), and head and neck location (66%). Due to small number of lymphoma events, no strong conclusions about prognostic ability of smIPI, COO, DPE, or DHL, could be made. S1001 demonstrated that 89% of pts maintained excellent outcomes after R-CHOP x 4 with PET-directed therapy. Only 11% of pts were iPET-pos and required radiation, but they also had excellent outcomes. Together with the FLYER trial in younger pts (Poeschel 2018), this NCTN trial establishes R-CHOP x 4 alone as the new standard approach to limited stage disease for majority of the pts. Disclosures Persky: Sandoz: Consultancy; Morphosys: Other: Member, Independent Data Monitoring Committee; Debiopharm: Other: Member, Independent Data Monitoring Committee; Bayer: Consultancy. Stephens:Acerta: Research Funding; Karyopharm: Research Funding; Gilead: Research Funding. Park:BMS: Consultancy, Research Funding; Rafael Pharma: Membership on an entity's Board of Directors or advisory committees; G1 Therapeutics: Consultancy; Teva: Consultancy, Research Funding; Gilead: Speakers Bureau; Seattle Genetics: Research Funding, Speakers Bureau. Bartlett:Seattle Genetics: Research Funding; ADC Therapeutics: Consultancy, Research Funding; Pfizer: Research Funding; Millenium: Research Funding; Bristol-Myers Squibb: Research Funding; Celgene: Research Funding; Genenetech: Research Funding; Immune Design: Research Funding; Gilead: Research Funding; Forty Seven: Research Funding; Janssen: Research Funding; Affimed: Research Funding; Merck: Research Funding; Pharmacyclics: Research Funding. Swinnen:Pharmacyclics: Consultancy; AbbVie: Consultancy. Barr:Celgene: Consultancy; Merck: Consultancy; Seattle Genetics: Consultancy; Genentech: Consultancy; Verastem: Consultancy; Gilead: Consultancy; Astra Zeneca: Consultancy, Research Funding; Janssen: Consultancy; AbbVie: Consultancy; Pharmacyclics LLC, an AbbVie company: Consultancy, Research Funding; TG Therapeutics: Consultancy, Research Funding. Leonard:Nordic Nanovector: Consultancy; Akcea Therapeutics: Consultancy; BeiGene: Consultancy; Gilead: Consultancy; Miltenyi: Consultancy; ADC Therapeutics: Consultancy; Nordic Nanovector: Consultancy; Akcea Therapeutics: Consultancy; Sandoz: Consultancy; ADC Therapeutics: Consultancy; Miltenyi: Consultancy; Genentech, Inc./F. Hoffmann-La Roche Ltd: Consultancy; Karyopharm Therapeutics: Consultancy; Epizyme, Inc: Consultancy; Sutro Biopharma: Consultancy; AstraZeneca: Consultancy; AstraZeneca: Consultancy; Bayer Corporation: Consultancy; Bayer Corporation: Consultancy; Celgene: Consultancy; Epizyme, Inc: Consultancy; Celgene: Consultancy; Genentech, Inc./F. Hoffmann-La Roche Ltd: Consultancy; Merck: Consultancy; MorphoSys: Consultancy; Karyopharm Therapeutics: Consultancy; Gilead: Consultancy; Sutro Biopharma: Consultancy; BeiGene: Consultancy; Merck: Consultancy; MorphoSys: Consultancy; Sandoz: Consultancy. Kahl:TG Therapeutics: Consultancy; ADC Therapeutics: Consultancy, Research Funding; Seattle Genetics: Consultancy; BeiGene: Consultancy. Fisher:Celgene: Consultancy; AstraZeneca: Consultancy; Barclays: Honoraria; prIME: Honoraria. Rimsza:NanoSting: Patents & Royalties: Named inventor on a patent licensed to NanoSting [Institution]. Smith:Portola Pharmaceuticals: Research Funding. Friedberg:Bayer: Honoraria, Other: Data & Safety Monitoring Committee; Acerta: Other: Data & Safety Monitoring Committee. OffLabel Disclosure: Ibritumomab tiuxetan in diffuse large B-cell lymphoma
A meta-analysis of genome-wide association studies of multiple myeloma among men and women of African ancestry
Du, Zhaohui , Weinhold, Niels , Song, Gregory, Rand, Kristin A. , Hwang, Amie E. , Sheng, Xin , Hom, Victor, Pawlish, Karen , Peters, Edward , Mohrbacher, Ann, Stram, Alexander , Berndt, Sonja I. , Blot, William J. , Stevens, Victoria L. , Goodman, Phyllis J. , Diver, W. Ryan , Hennis, Anselm , Nemesure, Barbara , Klein, Eric A. , Rybicki, Benjamin A. , Stanford, Janet L. , Witte, John S. , Signorello, Lisa B., John, Esther M. , Stroup, Antoinette M. , Stephens, Owen , Zangari, Maurizio , Hu, Jennifer J. , Nyante, Sarah J. , Carpten, John D. , Mehta, Jayesh, Wolf, Jeffrey L. , Martin, Thomas G. , Tomasson, Michael H. , Terebelo, Howard R. , Janakiraman, Nalini, Kolonel, Laurence N. , Auclair, Daniel , Chiu, Brian C.‐H. , Ziv, Elad , Vij, Ravi , Bernal‐Mizrachi, Leon , Morgan, Gareth J. , Zonder, Jeffrey A. , Huff, Carol Ann , Orlowski, Robert Z. , Conti, David V. , Haiman, Christopher A. , Orlowski, Robert Z. , John, Esther M , Klein, Eric A , Nyante, Sarah , Morgan, Gareth J , Ziv, Elad , Conti, David V , Berg, David Van Den , Ailawadhi, Sikander , Nooka, Ajay K. , Singhal, Seema , Bock, Cathryn Hufford , Stram, Alex , Casey, Graham , Kittles, Rick A. , Bernstein, Leslie , Rhee, Frits van , Olshan, Andrew F. , Zheng, Wei , Ziegler, Regina G. , Ingles, Sue A. , Press, Michael F. , Chanock, Stephen Jacob , Colditz, Graham A. , Fiala, Mark A. , Anderson, Kenneth C. , Marchand, Loı̈c Le , Stram, Daniel O. , Lonial, Sagar , Cozen, Wendy

Mayo Clinic

Blood Advances | January 14, 2020 | Volume: 4 | Issue: 1 | Pages: 181-190

10.1182/bloodadvances.2019000491 | PMID 31935283 Get It

Abstract Persons of African ancestry (AA) have a twofold higher risk for multiple myeloma (MM) compared with persons of European ancestry (EA). Genome-wide association studies (GWASs) support a genetic contribution to MM etiology in individuals of EA. Little is known about genetic risk factors for MM in individuals of AA. We performed a meta-analysis of 2 GWASs of MM in 1813 cases and 8871 controls and conducted an admixture mapping scan to identify risk alleles. We fine-mapped the 23 known susceptibility loci to find markers that could better capture MM risk in individuals of AA and constructed a polygenic risk score (PRS) to assess the aggregated effect of known MM risk alleles. In GWAS meta-analysis, we identified 2 suggestive novel loci located at 9p24.3 and 9p13.1 at P &lt; 1 × 10−6; however, no genome-wide significant association was noted. In admixture mapping, we observed a genome-wide significant inverse association between local AA at 2p24.1-23.1 and MM risk in AA individuals. Of the 23 known EA risk variants, 20 showed directional consistency, and 9 replicated at P &lt; .05 in AA individuals. In 8 regions, we identified markers that better capture MM risk in persons with AA. AA individuals with a PRS in the top 10% had a 1.82-fold (95% confidence interval, 1.56-2.11) increased MM risk compared with those with average risk (25%-75%). The strongest functional association was between the risk allele for variant rs56219066 at 5q15 and lower ELL2 expression (P = 5.1 × 10−12). Our study shows that common genetic variation contributes to MM risk in individuals with AA.
Distinct Cutoff Values of Adalimumab Trough Levels Are Associated With Different Therapeutic Outcomes in Patients With Inflammatory Bowel Disease
Park, Sang Hyoung , Kane, Sunanda V. , Papadakis, Konstantinos A. , Bruining, David H. , Faubion, William A. , Raffals, Laura E. , Pardi, Darrell S. , Tremaine, William J., Stephens, Michael C., Khanna, Sahil , Loftus, Edward V. , Loftus, Edward V , Al‐Bawardy, Badr , Aniwan, Satimai , Coelho–Prabhu, Nayantara , Kisiel, John B. , Willrich, Maria Alice V.

Mayo Clinic

Crohn's & Colitis 360 | November 26, 2019 | Volume: 1 | Issue: 3 | Pages: No page available

10.1093/crocol/otz047 Get It

Abstract Background and Aims We aimed to evaluate the relationship of serum adalimumab trough levels (ATL) with disease activity of inflammatory bowel disease (IBD) patients in a large, well-characterized referral center-based cohort. Methods We compared serum ATL between those with clinical, biochemical, or endoscopic/radiologic disease activity and those without. Results A total of 236 patients with IBD were included. Higher cutoff levels were associated with endoscopic and/or radiologic responses (cutoff value: 5.3 mcg/mL, P = 0.003) compared with improvement in C-reactive protein (cutoff value: 4.3 mcg/mL, P = 0.031). Conclusions Higher cutoff ATL was associated with endoscopic and/or radiologic response.
Disease Severity and Cost in Adhesive Small Bowel Obstruction
Finnesgard, Eric J. , Shariq, Omair A. , Knight, Ariel W. , Stephens, Daniel J. , Aho, Johnathon M. , Kim, Brian D. , Schiller, Henry J., Zielinski, Martin D. , Stephens, Daniel , Knight, Ariel, Hernandez, Matthew C. , Stephens, Daniel

Mayo Clinic

World Journal of Surgery | October 30, 2019 | Volume: 43 | Issue: 12 | Pages: 3027-3034

10.1007/s00268-019-05148-y | PMID 31555867 Get It

AbstractBackgroundAdhesive small bowel obstruction (ASBO) severity has been associated with important clinical outcomes. However, the impact of ASBO severity on hospitalization cost is unknown. The American Association for the Surgery of Trauma (AAST) developed an Emergency General Surgery (EGS) disease severity grading system for ASBO. We stratified patients’ ASBO severity and captured hospitalization costs hypothesizing that increased disease severity would correlate with greater costs.MethodsThis was a single‐center study of hospitalized adult patients with SBO during 2015–2017. Clinical data and estimated total cost (direct + indirect) were abstracted. AAST EGS grades (I–IV) stratified disease severity. Costs were normalized to the median grade I cost. Univariate and multivariate analyses evaluated the relationship between normalized cost and AAST EGS grade, length of hospital and ICU stay, operative time, and Charlson comorbidity index.ResultsThere were 214 patients; 119 (56%) were female. AAST EGS grades included: I (62%, n = 132), II (23%, n = 49), III (7%, n = 16), and IV (8%, n = 17). Relative to grade I, median normalized cost increased by 1.4‐fold for grade II, 1.6‐fold for grade III, and 4.3‐fold for grade IV disease. No considerable differences in patient comorbidity between grades were observed. Pair‐wise comparisons demonstrated that grade I disease cost less than higher grades (corrected p < 0.001). Non‐operative management was associated with lower normalized cost compared to operative management (1.1 vs. 4.5, p < 0.0001). In patients who failed non‐operative management, normalized cost was increased 7.2‐fold. Collectively, the AAST EGS grade correlated well with cost (Spearman's p = 0.7, p < 0.0001). After adjustment for covariates, AAST EGS grade maintained a persistent relationship with cost.ConclusionIncreasing ASBO severity is independently associated with greater costs. Efforts to identify and mitigate costs associated with this burdensome disease are warranted.Level of evidenceIII, economic/decision.
P2‐294: RETROSPECTIVE REVIEW OF CLINICAL FEATURES OF ABCA7 LOSS‐OF‐FUNCTION MUTATION CARRIERS IN LATE‐ONSET ALZHEIMER'S DISEASE
Stephens, Alana, Allen, Mariet , Atık, Merve, Lincoln, Sarah, Malphrus, Kimberly G., Nguyen, Thuy , Corda, M, Conway, Olivia J., Graff‐Radford, Neill R. , Ertekin‐Taner, Nilüfer , Corda, Morgane M., Dickson, Dennis W.

Mayo Clinic

Alzheimer's & Dementia | October 18, 2019 | Volume: 15 | Issue: 7S_Part_13 | Pages: No page available

10.1016/j.jalz.2019.06.2701 Get It

Factors That Influence Treatment Decision-Making: Perspectives of 1147 Chronic Lymphocytic Leukemia (CLL) Patients in the United States
Koffman, Brian , Dennison, Betsy, Kennard, Kaitlin, Byrd, John C. , Furman, Richard R. , Pagel, John M. , Nabhan, Chadi , Brander, Danielle M. , Hill, Brian T. , Ibarz, Javier Pinilla, Cheson, Bruce D. , Choi, Michael Y. , Henry, Thomas E, Mato, Anthony R. , Davids, Matthew S. , Kay, Neil E. , Siddiqi, Tanya , Wierda, William G. , Stephens, Deborah M. , Rosen, Steven T. , Awan, Farrukh T. , Roeker, Lindsey E.

Mayo Clinic

Blood | October 15, 2019 | Volume: 132 | Issue: Supplement 1 | Pages: 4414-4414

10.1182/blood-2018-99-112971 Get It

Abstract Introduction: Given the availability of several active treatment (tx) options for CLL, providers frequently make tx recommendations based on their interpretation of objective data from consensus criteria and clinical trials. There are limited data describing patient (pt) experience, values, and insights when encountering multiple tx choices. The CLL Society, a pt-driven, physician-curated nonprofit organization focused on the unmet needs of the CLL community, sought to explore how CLL pts make tx decisions. Methods: A 64 question survey was distributed online and in paper to CLL pts from Oct-Dec 2017. The survey was developed by CLL experts. The research was IRB-approved. Chi square was used for statistical comparison; all other analyses are descriptive in nature. Results: 1147 pts from 48 states completed the survey. Median age was 65 (range 28-86), 46% male, 96% Caucasian. Who Influences Tx: Of the 79% of pts who have seen a CLL expert, defined as a provider focused on CLL at an academic/research center, 95% rated their provider as very/extremely influential. 83% of pts rated their own opinions as influential, and 59% rated the opinion of a general hematologist/oncologist (gen heme/onc), a provider who sees a variety of cancer types, as influential. Only 27% let their provider unilaterally decide on tx. Of these pts, 19% trusted their provider to select the best tx and 5% didn't have enough understanding to be involved in the decision. 64% were influenced by outside sources of information, such as family members, live CLL support groups, or online sources including pt experts and forums. Factors in Deciding Tx: Of the 67% pts surveyed who have been treated, 73% were offered > 1 tx choice by their provider. In deciding on a specific tx, 94% of respondents stated that it was very/extremely important to know there were tx options beyond their current regimen. The most important factors in selecting tx were response rate (91%), overall survival (88%), progression-free survival (86%), long-term side effects (82%), ability to achieve undetectable MRD (80%), cost/insurance coverage (66%), and location of tx (50%) with significant differences between subgroups (Table 1). Tx was impacted by cost or insurance issues for 16% (n=104). Of these 104 pts, cost/insurance issues resulted in tx delay for 34 and change in tx plan for 24 pts. 16 pts reported that an appeal led to coverage and 16 pts received financial assistance. Pts' willingness to accept potentially curable but high-risk tx was heavily influenced by availability of alternate options. For example, 18% of pts would be willing to consider cytotoxic chemotherapy if alternate options were available vs. 72% if no options were available. Similar patterns were seen with CAR-T tx (28% vs. 79%) and bone marrow transplantation (BMT) (15% vs. 66%) with significant differences between subgroups (Table 2). 82% were willing to take a life-long tx for long-term control without potential for cure. Willingness to accept long-term tx was significantly higher in treated vs. untreated pts (84% vs. 77%, p=.006) and men vs. women (84% vs. 78%, p=.008). Clinical Trial Education: Of those treated by a gen heme/onc, 31% received no education on clinical trials. Only 15% reported good understanding of trial opportunities vs. 52% treated by a CLL expert (p<.001). 9% of pts treated by gen heme/onc were offered clinical trial participation vs. 47% treated by a CLL expert (p<0.001). Pts who declined or would have declined participation cited reasons including preference for "proven" tx (38%), distance from trial site (29%), fear (20%), frequent imaging (20%), overall lack of understanding about trials (15%), and lack of information (14%). Summary: Most respondents want to be and are involved in tx decisions. While providers are influential in decision making, CLL pts also rely on outside sources. Gender, age, and tx status influence which factors drive decision making. Pts select tx based on response rate, survival, side effects, and ability to achieve deep response and want a plan in place for sequencing tx. The majority are willing to consider long-term tx without cure. Few want chemotherapy, CAR-T, or BMT unless they have no other options. A significant opportunity for improvement in education on clinical trial opportunities was identified. This description of pt experience, values, and preferences enriches the informed consent process and may lead to more tailored and patient-centered care. Disclosures Kennard: AbbVie, Gilead, Verastem: Consultancy. Furman:TG Therapeutics: Consultancy; Loxo Oncology: Consultancy; Sunesis: Consultancy; Verastem: Consultancy; Incyte: Consultancy, Other: DSMB; Genentech: Consultancy; Acerta: Consultancy, Research Funding; Gilead: Consultancy; Janssen: Consultancy; Pharmacyclics LLC, an AbbVie Company: Consultancy; AbbVie: Consultancy. Pagel:Pharmacyclics, an AbbVie Company: Consultancy; Gilead: Consultancy. Davids:Sunesis: Membership on an entity's Board of Directors or advisory committees; Roche: Consultancy; Astra-Zeneca: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; BMS: Research Funding; Genentech: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Astra-Zeneca: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; MEI Pharma: Consultancy, Research Funding; Surface Oncology: Research Funding; Gilead: Membership on an entity's Board of Directors or advisory committees; Celgene: Consultancy; Merck: Consultancy; Merck: Consultancy; MEI Pharma: Consultancy, Research Funding; TG Therapeutics: Membership on an entity's Board of Directors or advisory committees, Research Funding; Astra-Zeneca: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; MEI Pharma: Consultancy, Research Funding; Abbvie: Consultancy, Membership on an entity's Board of Directors or advisory committees; Celgene: Consultancy; Sunesis: Membership on an entity's Board of Directors or advisory committees; Abbvie: Consultancy, Membership on an entity's Board of Directors or advisory committees; TG Therapeutics: Membership on an entity's Board of Directors or advisory committees, Research Funding; Genentech: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Pharmacyclics: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Gilead: Membership on an entity's Board of Directors or advisory committees; Verastem: Consultancy, Research Funding; Surface Oncology: Research Funding; Gilead: Membership on an entity's Board of Directors or advisory committees; Janssen: Consultancy, Membership on an entity's Board of Directors or advisory committees; Pharmacyclics: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Abbvie: Consultancy, Membership on an entity's Board of Directors or advisory committees; Celgene: Consultancy; Roche: Consultancy; Pharmacyclics: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Janssen: Consultancy, Membership on an entity's Board of Directors or advisory committees; BMS: Research Funding; Verastem: Consultancy, Research Funding; BMS: Research Funding; Genentech: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Surface Oncology: Research Funding; Verastem: Consultancy, Research Funding; TG Therapeutics: Membership on an entity's Board of Directors or advisory committees, Research Funding; Janssen: Consultancy, Membership on an entity's Board of Directors or advisory committees; Sunesis: Membership on an entity's Board of Directors or advisory committees; Merck: Consultancy; Roche: Consultancy. Nabhan:Cardinal Health: Employment, Equity Ownership. Kay:Acerta: Research Funding; Tolero Pharmaceuticals: Membership on an entity's Board of Directors or advisory committees, Research Funding; Janssen: Membership on an entity's Board of Directors or advisory committees; Morpho-sys: Membership on an entity's Board of Directors or advisory committees; Pharmacyclics: Membership on an entity's Board of Directors or advisory committees, Research Funding; Agios Pharm: Membership on an entity's Board of Directors or advisory committees; Celgene: Membership on an entity's Board of Directors or advisory committees; Gilead: Membership on an entity's Board of Directors or advisory committees; Infinity Pharm: Membership on an entity's Board of Directors or advisory committees; Cytomx Therapeutics: Membership on an entity's Board of Directors or advisory committees. Siddiqi:Juno Therapeutics: Other: Steering committee. Brander:Genentech: Consultancy, Honoraria, Other: Institutional research funding for non investigator initiated clinical trial, Research Funding; Pharmacyclics, an AbbVie Company: Consultancy, Honoraria, Research Funding; AbbVie: Consultancy, Honoraria, Other: Institutional research funding for non investigator initiated clinical trial, Research Funding; Teva: Consultancy, Honoraria; Novartis: Consultancy, Other: DSMB; TG Therapeutics: Consultancy, Honoraria, Other: Institutional research funding for non investigator initiated clinical trial, Research Funding; BeiGene: Other: Institutional research funding for non investigator initiated clinical trial, Research Funding; Acerta: Other: Institutional research funding for non investigator initiated clinical trial, Research Funding; DTRM: Other: Institutional research funding for non investigator initiated clinical trial, Research Funding. Wierda:AbbVie, Inc: Research Funding; Genentech: Research Funding. Hill:Abbvie: Honoraria, Membership on an entity's Board of Directors or advisory committees; Pfizer: Honoraria, Membership on an entity's Board of Directors or advisory committees; Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees; Seattle Genetics: Honoraria, Membership on an entity's Board of Directors or advisory committees; Genentech: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Pfizer: Honoraria, Membership on an entity's Board of Directors or advisory committees; Seattle Genetics: Honoraria, Membership on an entity's Board of Directors or advisory committees; Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees; Pharmacyclics: Honoraria, Membership on an entity's Board of Directors or advisory committees; Amgen: Research Funding; Abbvie: Honoraria, Membership on an entity's Board of Directors or advisory committees; Pharmacyclics: Honoraria, Membership on an entity's Board of Directors or advisory committees. Cheson:AbbVie, Roche/Genentech, Pharmacyclics, Acerta, TG Therapeutics: Consultancy. Choi:Pharmacyclics: Consultancy, Research Funding, Speakers Bureau; Gilead: Speakers Bureau; Rigel: Consultancy; Genentech: Speakers Bureau; AbbVie, Inc: Consultancy, Speakers Bureau. Mato:AstraZeneca: Consultancy; Prime Oncology: Honoraria; Portola: Research Funding; Johnson & Johnson: Consultancy; Medscape: Honoraria; Celgene: Consultancy; TG Therapeutics: Consultancy, Research Funding; Acerta: Research Funding; Pharmacyclics, an AbbVie Company: Consultancy, Research Funding; AbbVie: Consultancy, Research Funding; Regeneron: Research Funding.