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Background Engraftment syndrome (ES) is a common complication of autologous hematopoietic cell transplantation (HCT). The difference in incidence of ES between melphalan formulations has not been widely reported throughout the literature and would allow for a more comprehensive understanding of the advantages and disadvantages of both melphalan formulations. Patients and methods This retrospective, single-center, observational study evaluated 83 adult multiple myeloma and immunoglobulin light chain amyloidosis patients who received either propylene glycol-containing (PG) or propylene glycol-free (PG-free) melphalan 140 mg/m2 as single-agent conditioning chemotherapy for autologous HCT from May 31, 2015 to May 31, 2019. The primary outcome was to assess the incidence of ES, as defined using the Maiolino criteria, with both melphalan formulations. Secondary outcomes included an analysis of potential risk factors for the development of ES, as well as an evaluation of overall length of stay (LOS). Results The incidence of ES for PG and PG-free melphalan did not differ significantly, 14/39 (35.9%) and 12/44 (27.3%) (P = 0.4), respectively. No potential risk factors for ES were identified on multivariate logistic regression analysis. A statistically significant difference in number of days to engraftment was identified for PG and PG-free melphalan, 15.56 vs. 13.82 days (P = 0.01), respectively; although, this did not translate to a decrease in LOS, 19.9 vs. 18.59 days (P = 0.14). Conclusions The incidence of ES did not differ significantly between melphalan formulations. Future research is needed to determine whether the faster time to engraftment seen with PG-free melphalan may translate to a decrease in LOS.

Use of respondent records is a method for supplementing survey data. Records can improve data quality when data collected by self-report are subject to recall or other measurement errors. One limitation of using respondent records is that respondents often cannot provide records for all survey items of interest. We explored the availability of respondent records using data from the Consumer Expenditure Records Study (CE Records Study). The CE Records Study is a non-probability feasibility study that examined the accuracy of self-reported data for various types of household expenditures. In the first interview, participants provided self-reports about the cost of household expenditures from the previous three months. In a follow-up interview, participants provided records (e.g., receipts, bank statements, bills) for all expenditures asked about. By comparing self-reports and records, we were able to evaluate the accuracy of self-reports. Records were available for 36% of the 3,039 expenditures reported. There were several expenditure and respondent characteristics that were associated with the availability of records suggesting that use of retrospective record collection may be more successful for supplementing self-reported data for certain types of consumer surveys compared to others.

Paper and web surveys often include grid-style questions formatted to save space and avoid repetition. However, this format is often discouraged by methodologists because of data quality concerns, particularly when respondents are using small screens (e.g., smartphones). In the World Trade Center Health Registry’s Wave 4 survey, we used grids to maintain comparability with prior waves. However, due to the rising number of respondents using smartphones to complete web surveys, we used responsive web design programming to automatically reformat grids into a series of individual items when a small screen device was detected. This method allowed us to retain grids used in previous years, yet address the issue of grids displaying poorly on mobile devices. We compared indicators of data quality (e.g., missing data, straight-lining) across grid formats to see whether the smartphone-optimized version suggests poorer, equal, or better data quality than the traditional grid. We also compared consistency with data collected in previous waves of the survey. We found some evidence that the optimized grid format improved data quality, and the benefits we observed may even suggest that some variant of the mobile-optimized format should be considered for all devices, regardless of screen size.

Self-reports of financial information in surveys, such as wealth, income, and assets, are particularly prone to inaccuracy. We sought to improve the quality of financial information captured in a survey conducted by phone and in person by encouraging respondents to check records when reporting on income and assets. We investigated whether suggestive prompts influenced unit response, compliance with the request to check records, precision of estimates, and accuracy. We conducted a split sample experiment in the Community Advantage Panel Survey in which half of telephone respondents and half of in-person household interview respondents were encouraged to check the records. We found a modest positive effect of prompts on compliance but no effect on unit response, precision, or accuracy.

Twitter is an online microblogging tool that disseminates more than 400 million messages per day, including vast amounts of health information. Twitter represents an important data source for the cancer prevention and control community. This paper introduces investigators in cancer research to the logistics of Twitter analysis. It explores methodological challenges in extracting and analyzing Twitter data, including characteristics and representativeness of data; data sources, access, and cost; sampling approaches; data management and cleaning; standardizing metrics; and analysis. We briefly describe the key issues and provide examples from the literature and our studies using Twitter data to understand public health issues. For investigators considering Twitter-based cancer research, we recommend assessing whether research questions can be answered appropriately using Twitter, choosing search terms carefully to optimize precision and recall, using respected vendors that can provide access to the full Twitter data stream if possible, standardizing metrics to account for growth in the Twitter population over time, considering crowdsourcing for analysis of Twitter content, and documenting and publishing all methodological decisions to further the evidence base.

Summary. A serious adverse effect that can limit the utility of ifosfamide is neurotoxicity, known as ifosfamide-induced encephalopathy (IIE). Information regarding the usefulness of prophylactic administration of methylene blue, thiamine, and albumin to reduce the incidence of IIE is scarce. We present the results of a retrospective chart review evaluating the incidence of IIE in adult patients who received ifosfamide to treat sarcoma at the University of Washington within a 2-year period. Patients who received methylene blue, thiamine, and/or albumin were compared to patients who did not receive these prophylactic agents concurrently with ifosfamide. The primary objective was to evaluate if prophylaxis is associated with a reduced incidence of IIE. Identifying risk factors associated with IIE was a secondary objective. The cost of using prophylaxis with ifosfamide administration was reviewed. Results. A total of 166 cycles were included. For the primary endpoint, more cycles in the prophylaxis group had patients with symptoms of IIE (21.1% in the prophylaxis group vs. 8.4% in the nonprophylaxis group); p = 0.026. The average number of risk factors per cycle was the same in each group; however, the type of risk factors differed. Conclusions. Prophylaxis was not associated with a reduced incidence of IIE, and it does not appear to delay time to onset of symptoms at our institution. Results of this study provide no support for using methylene blue, thiamine, and/or albumin as routine prophylaxis when administering ifosfamide to adults with sarcoma.

Prognosis of elderly patients with acute myeloid leukemia (AML) remains poor and new treatment approaches are urgently needed. A novel nucleoside analog sapacitabine has recently emerged as a feasible agent because of its oral administration and acceptable toxicity profile. Clinical efficacy of sapacitabine, both as a single agent and in combination, has been evaluated in elderly AML patients or AML patients unfit for standard intensive chemotherapy. Response rates varied from 15 to 45% in phase II studies. Sapacitabine was overall well-tolerated with gastrointestinal and myelosuppression-related complications were the most common side effects. Unfortunately, in a phase III study sapacitabine showed no clinical superiority as compared to low-dose cytarabine (LDAC) in patients with AML. Another large phase III study comparing the combination of sapacitabine with decitabine to decitabine alone is currently ongoing and is expected to be completed by the end of 2015 or by the first half of 2016.

Argininosuccinicaciduria is a rare metabolic disorder of the urea cycle associated with the inability to excrete nitrogenous waste in the form of urea. Along with low serum arginine, hepatomegaly, and mental retardation, congenital trichorrhexis nodosa is a distinguishing feature of the disorder. We present a 3.5-year-old girl diagnosed with argininosuccinicaciduria who presented to the dermatology clinic with hair thinning and loss since birth. Microscopic evaluation revealed nodular swellings on the hair shafts and frayed cortical fibers consistent with the diagnosis of trichorrhexis nodosa occurring in the setting of argininosuccinicaciduria.

Purpose:This study examined individual contributions to overall pacing strategy during 2- and 5-km rowing trials in a coxless-4 boat.Methods:A crew of 4 male rowers performed maximal-effort on-water trials over 2 and 5 km, and power output during every individual stroke was measured for each crew member. Mean overall boat and individual rower stroke power were calculated for each 25% epoch (25% of total strokes taken), and power for each individual epoch was calculated as a percentage of mean power maintained over the entire distance. The coefficient of variation was used to determine stroke-to-stroke and epoch-to-epoch variability for individual rowers and the overall boat.Results:In both trials, the overall pacing strategy consisted of a high power output in the initial 25% that decreased in the middle 50% and increased again in the final 25%. However, individual rower data indicate wide variation in individual power profiles that did not always mimic the overall boat profile.Conclusions:This study demonstrates that overall boat power profiles during 2- and 5-km rowing trials are similar to velocity profiles previously reported for individual ergometry and on-water racing events. However, this overall profile is achieved despite considerable variation in individual rower profiles. Further research is warranted to determine the mechanisms through which individual contributions to overall pacing strategy are regulated and the effectiveness or otherwise of seemingly disparate individual strategies on overall performance.

Abstract 4301 Background: Currently, there is no standard reinduction regimen for relapsed and refractory (RR) AML. In order to evaluate responses to reinduction regimens we present a retrospective side-by-side comparison frequently used AML salvage regimens at our institution: CECA, HiDAC and CLAG-M. Method: From April 2007 to May 2011, 74 consecutive patients with RR AML received CECA (n=44), HiDAC (n=18) or CLAG-M (n=12). The primary outcome was complete remission (CR) rates and secondary outcomes were overall survival (OS) and relapse free survival (RFS). Additional variables such as toxicity and transplant data were also examined. Result: Baseline characteristics among the three groups were similar except for relapse status, with a greater proportion of AML patients receiving CECA after first relapse and HiDAC and CLAG-M after multiple relapses (Table 1). The mean age was 55.6 (range, 24–70), 53.8 (range, 25–71) and 49.5 (range, 23–68) years for CECA, HiDAC and CLAG-M respectively (P=0.38). For CECA, HiDAC and CLAG-M, respectively, 29.5%, 27.7% and 50% of patients were classified with therapy-related AML (P=0.85) and 11.4%, 11.1% and 16.7% of patients were classified with AML transformed from MDS (P=0.62). There was no difference in cytogenetic risk groups between the cohorts with the majority of the patients classified as intermediate risk. The mean blast percentage at the start of treatment was equivalent between the three groups at 39.8%, 27.4% and 26.7% for CECA, HiDAC and CLAG-M respectively (P=0.25). Median follow-up for all patients was 3.5 months. CR rates for CECA, HiDAC and CLAG-M were similar (20.5%, 16.7%, 41.7%; P=0.44). Median OS was 5 months for patients receiving CECA, 5 months for HiDAC and 3.5 months for CLAG-M (P=0.91) (Figure 1A). RFS was also similar among the three groups (p=0.91) (Fig. 1B). Of patients treated with CECA, HiDAC and CLAG-M, 27.2%, 38.8% and 41.6% proceeded to allogeneic hematopoietic cell transplant (P=0.71). Of the 8 patients who achieved CR with CECA but did not proceed to transplant, 5 patients had a CR of less then 3 months, 1 refused further care, 1 was lost to follow up and 1 received further consolidation therapy then relapsed. The mean length of stay in the hospital was 33.6±3.4, 99.7±60.3 and 45.4 ±35.7 days respectively for CECA, HiDAC and CLAG-M (P=0.19). The 30-day treatment related mortality rate was 2.3%, 16.7% and 16.7% respectively (P=0.13). Patients who received CECA had a higher number of total adverse events than patients who received HiDAC and CLAG-M (Table 1), with higher rates of pulmonary edema, sepsis, fungal pneumonia and C. difficle colitis. However, due to the small number of patients per cohort, only descriptive statistics are reported. Conclusion: CECA, HiDAC and CLAG-M are equivalent AML salvage regimens in terms of CR, OS, and RFS. Although each resulted in different adverse event profiles. These data provide a basis for designing prospective clinical studies and sample size estimates to formally compare three common reinduction regimens in RR AML. Disclosures: No relevant conflicts of interest to declare.

Graft-versus-host-disease (GVHD) is a life threatening complication of allogeneic hematopoietic stem cell transplantation (HSCT). The calcineurin inhibitor tacrolimus is commonly used in combination with other immunosuppressants to prevent GVHD; however the optimal serum target concentration for tacrolimus in this population remains unknown. A retrospective review was conducted to determine whether an association exists between tacrolimus concentrations and transplant-related outcomes, specifically acute GVHD, as well as the rate of renal toxicity and mortality. Data from 203 patients who underwent an allogeneic HSCT from a related (n1⁄495) or unrelated (n1⁄4108) donor between January 1, 2003 and December 31, 2011 at a large academic hospital was analyzed. Sixty-two (31%) patients developed acute GVHD within the first 30 days following allogeneic stem cell transplant. Median tacrolimus concentrations at day 0, 7, 14, and 28 were 11.2, 13.8, 13.5 and 10.2 ng/mL, respectively among those who developed acute GVHD. Patients who did not develop acute GVHD had similar tacrolimus concentrations of 10.4,13.1,12.3 and 9.9 ng/mL for the same respective time points. Serum tacrolimus concentrationswere similar across all grades of GVHD and therewas no correlation between drug concentrations with respect to renal toxicity. Patients who developed renal dysfunction (n1⁄418) had tacrolimus concentrations similar to the median concentration of the entire cohort, including those who did not experience renal toxicity. Overall mortality among the 203 included patients was 49%. Mortality was higher among patients who developed acute GVHD (55%) than in patients who did not experience the disease (47%). The results of this analysis support previous conclusions that tacrolimus blood concentrations are not associated with acute GVHD within the first 30 days post allogeneic HSCT.