Utility Of Stem Cell Transplantation Research Articles

Factors determining utilization of stem cell transplant for initial therapy of multiple myeloma by patient race: exploring intra-racial healthcare disparities

Multiple myeloma (MM) therapeutics have evolved tremendously in recent years, with significant improvement in patient outcomes. As newer treatment options are developed, stem cell transplant (SCT) remains an important modality that provides excellent disease control and delays the progression of disease. Over the years, SCT use has increased overall in the U.S., but two distinct gaps remain, including suboptimal use overall and racial-ethnic disparities. We evaluated the National Cancer Database (NCDB) to study what sociodemographic factors might play a role within a given racial-ethnic group leading to disparate SCT utilization, such that targeted approaches can be developed to optimize SCT use for all. In nearly 112,000 cases belonging to mutually exclusive categories of non-Hispanic Whites (NHW), non-Hispanic Blacks (NHB), Hispanics, non-Hispanic Asians (NHA), and others, we found certain factors including age, comorbidity index, payor type, facility type (academic vs. community) and facility volume to be uniformly associated with SCT use for all the racial-ethnic groups, while gender was not significant for any of the groups. There were several other factors that had a differential impact on SCT utilization among the various race-ethnicity groups studied, including year of diagnosis (significant for NHW, NHB, and Hispanics), income level (significant for NHW and Hispanics), literacy level (significant for NHW and NHB), and geographic location of the treatment facility (significant for NHW and NHA). The suboptimal SCT utilization overall in the U.S. suggests that there may be room for improvement for all, even including the majority NHW, while we continue to work on factors that lead to disparities for the traditionally underserved populations. This study helps identify sociodemographic factors that may play a role specifically in each group and paves the way to devise targeted solutions such that resource utilization and impact can be maximized.

With Equal Access, African Americans with Non-del17p Multiple Myeloma Have Superior Overall Survival, but del17p Still Carries Poor Prognosis across Race: A VA Study

BACKGROUND: In multiple myeloma (MM), deletion of TP53/17p (del17p), present in around 10% of patients, is associated with shortened survival. Lower incidence of del17p is reported in African Americans (AA) compared to European Americans (EA), alluding to possible contribution of disease biology to racial differences in outcome among AA and EA patients with MM. Our recent report of a significantly superior age-adjusted risk of death in AA compared to EA patients in the younger (<65) Veteran population also suggests that AA may have a genetic predisposition that renders them to have better therapeutic outcome or have more indolent subtypes of MM. Here we investigated the incidence and impact of del17p on outcome in AA and EA patients with MM at the VA. METHODS: We identified 2677 patients with MM from 1999 to 2017 using the VA's nationwide Corporate Data Warehouse for whom information on del17p was available. We extracted data on patients' age, race, ISS stage, therapy at induction and stem-cell transplant (SCT) utilization. Test results for del17p were extracted from lab panel and pathology reports. Our analysis is focused on patients with this data. RESULTS: Of the 2677 MM patients evaluated for del17p by conventional cytogenetics and/or FISH, self-reported race information was available in 2432 patients, among which AA constituted 35.6% (867) and EA 64.4% (1565). AA had a greater proportion of younger (<65) patients compared to EA (49.48% vs 33.87%, p<0.001). Overall, among those tested, del17p was reported in 7.4% of all patients, but the incidence was significantly lower in AA compared to EA (4.73% vs 8.82%, p<0.001), largely due to the difference in incidence of del17p in younger AA vs EA (3.73% vs 8.30%, p = 0.005). The rates of del17p across different ISS stages were not significantly different, nor did ISS stage differ across race. As expected, del17p was associated with shortened survival. In the full cohort, median survival was 2.26 years for patients with del17p and 4.27 years for those without (p<0.001). These differences were also observed within race (1.74 vs 4.95 in AA, p<0.001, 2.34 vs 4.13 in EA, p<0.001) and age groups (2.67 vs 5.49 in younger, p<0.001; 2.12 vs 3.5 in older, p<0.001). We found no significant racial differences in survival between AA and EA patients with del17p deletion (1.74 vs 2.34, p=0.47) regardless of age category. However, importantly we noted a significant racial difference in the median survival between younger AA and EA without del17p (7.75 vs. 4.87 years, p=0.006). In contrast, in older patients without del17p we observed no significant difference in survival across race. To understand the difference in survival between younger AA and EA without del17p, we compared ISS stage, ECOG status, and novel therapy (bortezomib, lenalidomide, and thalidomide) and SCT utilization between these groups. No significant differences were observed apart from a small difference in utilization of bortezomib in AA patients as compared to EA at induction (63.1% vs 58.3%, p = 0.029). To assess whether bortezomib utilization had any impact on survival, we compared survival among younger AA and EA without del17p after stratifying into three treatment regimens: bortezomib/dex (Vd), lenalidomide/dex (Rd), and RVd. We observed superior survival for AA as compared to EA across all three regimens (Vd: 5.82 vs 3.63 years, p=0.022; Rd: 8.15 vs 5.10, p=0.029; RVd: median not reached vs 6.95, p=0.72), suggesting that the difference in bortezomib utilization was not responsible for the observed difference in survival. Interestingly, among 198 patients with del17p for whom the percentage of cells with deletion was reported, we observed that higher (>55%) clonality was associated with lower median survival compared to those with low clonality (5-55%) (0.97 vs 2.42, p=0.049). The difference was even more significant with a threshold of 35% (p=0.015). CONCLUSIONS: Our large study identified significantly lower incidence of del17p in younger AA compared to EA. We found no racial disparity in survival among AA and EA with del17p regardless of age. In contrast, in the absence of del17p, younger AA demonstrated significantly better survival as compared to younger EA and better response to bortezomib- and lenalidomide-based doublet and triplet induction regimens, suggesting possible differences in disease biology other than del17p that may contribute to a more indolent course and increased sensitivity to therapy. Disclosures Yellapragada: Celgene: Research Funding; Novartis: Employment, Other: Spouse Employment ; BMS: Research Funding; Takeda: Research Funding. Munshi:Takeda: Consultancy; Janssen: Consultancy; Oncopep: Consultancy; Takeda: Consultancy; Oncopep: Consultancy; Celgene: Consultancy; Janssen: Consultancy; Celgene: Consultancy; Amgen: Consultancy; Amgen: Consultancy; Abbvie: Consultancy; Abbvie: Consultancy; Adaptive: Consultancy; Adaptive: Consultancy.

Factors Determining Utilization of Stem Cell Transplant (SCT) for Initial Therapy of Multiple Myeloma (MM) By Patient Race: Exploring Intra-Racial Healthcare Disparities

Background: SCT is a standard therapeutic approach for patients with MM and has contributed to improved outcomes. While SCT utilization in MM has increased for all racial-ethnic subgroups over time, population-based studies have repeatedly shown that racial minorities are less likely to receive it. We explored factors that determine SCT utilization among patients within a given race to understand intra-racial disparities and how to address them in order to maximize the use of this imperative treatment among eligible patients.Methods: The 2016 National Cancer Database (NCDB) for MM with patients diagnosed between 2004-2013 was studied. Cases without information on the selected patient or facility characteristics evaluated in this analysis, including SCT use, race, median income, insurance status or facility type of initial MM diagnosis and/or treatment were excluded. The association between utilization of SCT as part of initial therapy of MM and socio-demographic factors was studied stratifying patients into mutually exclusive racial-ethnic subgroups non-Hispanic Whites (NHW), non-Hispanic Blacks (NHB), Hispanics, non-Hispanic Asians (NHA) and others using multivariate GEE logistic regression analysis (clustered on facility ID). Interactions between race and patient/facility characteristics and SCT utilization were tested using similar methods but including all races.Results: Of the 123480 unique MM patients in NCDB diagnosed between 2004-2013, 111799 met the inclusion criteria. Of these, 15021 patients (13.4%) received SCT as part of initial MM therapy. Of those who received SCT, 77.5% were NHW (14.4% of all NHW), 15.1% NHB (10.3% of all NHB), 5.2% Hispanics (13.2% of all Hispanics), 2% NHA (15.5% of all NHA) and <1% others. There was no difference by gender but significantly reduced odds of receiving SCT were noted for every 10-year increase in age at diagnosis for all racial subgroups. NHA were the only subgroup not to have a significant year-wise increase over time in odds of receiving SCT between 2004-2013. A significant increase in SCT utilization was seen with increasing median income (quartiles; 2008-2012 data), for NHW and Hispanics but not for NHB and NHA, while increased education (% population with high-school degree; 2008-2012 data) was associated with a significant increase in SCT use for NHW and NHB, but not among Hispanics or NHA. An increase in great circle distance (GCD) from the treating facility as well as a lower Charlson-Deyo comorbidity index (CCI) was associated with significantly increased odds of receiving SCT for all racial groups. Insurancepayer status significantly affected odds of receiving SCT among all races with those having private insurance to be most likely to receive SCT among NHW, NHB and Hispanics while those with “other government” insurance payer type being most likely among NHA. Among treating facility characteristics, patients of all races were more likely to receive SCT if treated at an academic/researchfacility (including NCI-designated centers) or centers with highest quartile of patientvolume seen. For analysis by geographicalregion, NHW and NHA had the highest odds of receiving SCT in mountain region but there was no impact of geography on SCT for NHB or Hispanics. Odds ratio (OR) of receiving SCT as part of initial MM therapy by socio-demographic and facility characteristics for patients of individual racial subgroups are shown in Table 1. A significant interaction between odds of receiving SCT and various racial subgroups was noted for patient age at diagnosis (p<.001, decreased odds among NHW and NHA), GCD from treating facility (p=0.013, increased odds among NHB and NHA), CCI (p=0.043, decreased odds among NHA) and treatment facility volumes (p=0.046, increased odds among NHA and Hispanics).Conclusions: In the largest analysis exploring socio-demographic factors affecting SCT utilization in MM we note a significant disparity within races for who gets SCT as a part of initial MM management and who does not. While some of these factors are non-modifiable, others including access to healthcare, income, insurance and literacy levels, proximity to treatment center, treating facility type as well as volumes are modifiable and appear to be barriers to SCT among in some races, while not in others. These must be addressed to minimize disparities within racial subgroups, especially the minorities and realize maximal benefit of SCT for MM. [Display omitted] DisclosuresAilawadhi:Novartis: Consultancy, Honoraria; Pharmacyclics: Research Funding; Amgen: Consultancy, Honoraria; Takeda: Consultancy, Honoraria. Sher:LAM Therapeutics, Inc: Research Funding.

HLA-mismatched bone marrow transplantation in severe aplastic anemia

Hematopoietic stem cell transplantation (SCT) can be curative for patients with severe aplastic anemia (SAA).1, 2 Concerns about graft failure and graft-versus-host disease (GVHD), more so for human leukocyte antigen (HLA) -mismatched donors, have impeded the utility of SCT for these patients. The introduction of post-SCT cyclophosphamide (PTCy) has been shown to decrease alloreactivity in the host-versus-graft and graft-versus-host directions and could be particularly well suited for SAA patients.3 Only a few patients in two recent reports have documented outcomes of patients with SAA treated with a haploidentical transplant and PTCy-based GVHD prophylaxis, both using non-myeloablative (NMA) conditioning with fludarabine, cyclophosphamide and 2 Gy total body irradiation (TBI).4, 5 Concern for higher rejection rate, both primary and secondary, has prompted us to use a more intense conditioning regimen, as previously described.6 Here, we report clinical outcomes in transfusion dependent (n=6, 5 were refractory to first line immunosuppressive therapy, one patient received hypomethylating agent for hypoplastic MDS subsequently diagnosed as AA) SAA patients who underwent HLA-mismatched bone marrow SCT from haploidentical (n=2) and 9/10 matched unrelated donors (n=4) at our center between January 2010 and May 2013. Conditioning regimen consisted of melphalan (140 mg/m2) on day-8, thiotepa (5 mg/kg) on day-7 and fludarabine (160 mg/m2) on days −6 to −3. GVHD prophylaxis consisted of cyclophosphamide (50 mg/kg) on days +3 and +4, tacrolimus for 180 days and mycophenolate mofetil for 100 days post transplant. Median age of patients at the time of SCT was 37 years (range 21–50), median Karnofsky performance score was 90 (range 80–100) and median time from diagnosis to transplant was 9 months (range 2–15 months). Of the six patients, two of them had a concomitant PNH clone>20% prior to SCT. All patients engrafted the donor cells with 100% chimerism noted for both myeloid and T-cell lineage at day +30 post SCT. Of the four-long-term survivors, 100% donor (lymphoid and myeloid) chimerism was noted starting from day 30 until 1 year post SCT in three patients and for up to 6 months for one patient (follow-up care was transferred to an outside institution and was found to be disease-free at 1 year post SCT). Median time to neutrophil and platelet engraftment was 16 and 13 days, respectively (Table 1). Acute GVHD was seen in three patients (grades-1, 2 and 4 each) and led to death in one patient who had a 9/10 MUD SCT (Table 1). No chronic GVHD (cGVHD) or disease relapse was noted. After a median follow-up of 949 days (range 735–1617), 4/6 (66.6%) patients (two haploidentical and two 9/10 MUD) were alive, fully engrafted without cGVHD.

The Real-World Characteristics and Outcomes of Newly Diagnosed Myeloma Patients Ineligible for Clinical Trials

Background:Over the past 2 decades, treatment advances have greatly improved the outcomes of patients with multiple myeloma (MM) and a glut of new knowledge about the biology, genetic profile, and prognostic factors of the disease have become available. However, much of this research has excluded patients with low performance status and those with impaired renal function or marrow reserve, therefore, relatively little is known about these patients who are largely ineligible for clinical trials. We performed a secondary data analysis of the Multiple Myeloma Research Foundation (MMRF) CoMMpass study to compare the disease manifestation, treatment, and prognosis of these patients with the overall MM population.Methods: Data was extracted from the open-access MMRF Researcher Gateway corresponding with interim analysis 8 from the CoMMpass study. The CoMMpass study is enrolling 1000 newly diagnosed MM patients who will be tracked longitudinally for 5 years. CoMMpass collects relevant clinical data as well as sequential tissue samples. Eligibility requirements for CoMMpass include: symptomatic MM with measureable disease by SPEP (≥1.0g/dL), UPEP (≥200mg/24 hours), or SFLC (≥10mg/dL); receiving an immunomodulator (IMID) and/or a proteasome inhibitor (PI) for initial MM treatment; and no prior malignancies in the past 5 years.We defined ineligibility for clinical trials as one or more of the following at time of MM diagnosis: ECOG performance status 3 or 4; creatinine ≥ 2.0mg/dL or receiving dialysis; absolute neutrophil count (ANC) <1.0x109/L or receiving growth factor support; or platelet count <50,000 x109/L or receiving platelet transfusion support. We compared the demographics, baseline presentation (International Staging System [ISS] stage, marrow and extramedullary disease burden, and chromosomal abnormalities), and first-line treatment of these patients with the overall population using bivariate analyses. We then performed a multivariate Cox regression analysis to compare event-free survival (EFS) defined as the interval from diagnosis to disease progression or death. The level of significance was set at 0.05 for all tests.Results:799 patients were available for analysis. The median age was 64 years, 61% (486) were male, and 76% (611) were white. 23% (180) met the study definition of ineligibility for clinical trials. 5% (41/778) had low performance status, 16% (128/799) impaired renal function, 3% (22/799) impaired marrow reserve.Patients ineligible for clinical trials were similar to the rest of the population demographically, but were more likely have higher risk disease by ISS (70% stage 3 compared to 20%, p <0.0001), and deletion of 17p (11% compared to 6%, p <0.0298), and were less likely to receive IMIDs during first-line therapy (35% compared to 64%, p <0.0001). Interestingly, stem cell transplant (SCT) utilization was similar between the two groups (32% compared to 34%, p >0.05). Bivariate analyses are summarized in Table 1.After controlling for ISS stage, deletion of 17p, first-line treatment, and SCT utilization, patients ineligible for clinical trials had a 56% (aHR 1.56 [95% CI 1.09- 2.23], p = 0.0141) increase in risk of disease progression or death.Conclusion: Newly diagnosed MM patients with low performance status, impaired renal function, or marrow reserve have long been considered a high-risk group and, thus, most clinical trials have excluded them. IMIDs are contraindicated for these patients due to its myelotoxicity which further limits treatment options. After adjusting for differences in treatment, these patients still have poorer outcomes. Additional research is needed to better understand the needs of these patients and improve their outcomes. [Display omitted] DisclosuresVij:Karyopharma: Consultancy; Bristol-Myers Squibb: Consultancy; Takeda: Consultancy, Research Funding; Amgen: Consultancy, Research Funding; Celgene: Consultancy; Janssen: Consultancy; Novartis: Consultancy; Jazz: Consultancy; Shire: Consultancy.

Racial Disparities in Treatment Utilization for Multiple Myeloma

Background: Recent treatment advances have greatly improved the prognosis of patients with multiple myeloma (MM). However, the utilization of some of these newer treatments is still low, particularly among black patients. Researchers initially attributed this disparity to access barriers such as lower socioeconomic status (SES) and/or inadequate health insurance. However, my colleagues and our group recently reported that black patients treated at our institution were 54% less likely to undergo stem cell transplantation (SCT) after controlling for these potential access barriers (Fiala, et al, BBMT, 2015).In this study, we aimed to confirm our previous finding on racial disparities in SCT utilization using the Surveillance, Epidemiology, and End Results (SEER)-Medicare linked database. We also reviewed the utilization patterns of bortezomib (BTZ), a topic not reported on to date. We chose BTZ as a comparator as it has largely become the standard of care in recent years and, unlike SCT, BTZ is not exclusive to specialized centers.Methods: All MM cases in the SEER-Medicare dataset from 2000-2011 were reviewed along with their corresponding claims data through 2013. We excluded cases with incomplete records such as: death certificate or autopsy cases, cases not enrolled in Medicare Part A and Part B, or did not have >1 claim one year prior to diagnosis, and managed care (HMO) enrollees; and cases where reported race was not white or black or where MM was diagnosed prior to age 65.The presence or absence of the corresponding procedure codes for SCT and BTZ were used to determine their utilization. Multivariate logistic regression was performed to analyze the impact of race on treatment utilization controlling for baseline health measures (age at diagnosis, performance status [PS] indicators, and Charlson Comorbidity Index [CCI] Score) and potential access barriers (SES [measured by the median household income (MHI) of census tract of home residence], Medicaid enrollment, and urban/rural status).The analysis of SCT utilization was limited to patients < 78 years old at diagnosis as coverage for SCT in older patients was previously restricted; BTZ utilization to patients diagnosed after 2003 to coincide with its FDA approval. We then performed multivariate Cox regression to analyze how the utilization patterns of these two treatments impact overall survival.Results: A total of 11,269 patients were included in the analysis of SCT utilization and 16,037 for BTZ utilization. The 8,625 patients included in both were included in the survival analysis.Overall, 84% were white and 16% were black. Black patients were younger at diagnosis (75.9 years compared to 77.3 years, p < 0.0001), but were more likely to have indicators of poor PS (27% compared to 19%; p < 0.0001), and a CCI > 1 (59% compared to 47%; p < 0.0001). Black patients also had lower SES on average (MHI $35,500 compared to $52,400; p < 0.0001) and were more likely to be Medicaid beneficiaries (34% compared to 11%, p < 0.0001).After controlling for overall health and potential access barriers, black patients were 37% (aOR = 0.63 [95% CI 0.49-0.80], p < 0.0001) less likely to utilize SCT, and 21% (aOR 0.79 [95% CI 0.72-0.88], p< 0.0001) less likely to utilize BTZ. The underutilization of these agents was the associated with a 12% (aHR 1.12 [95% CI 1.05-1.19], p = 0.0007) increase in hazard ratio for death among black patients.Conclusion: Eliminating health disparities is a current focus of U.S. public policy. This study highlights the complexity of the issue. In patients with MM, treatment disparities are not completely explained by poorer baseline health or increased access barriers among black patients. Differences in individual decision-making among black and white patients may explain part of the disparity. DisclosuresNo relevant conflicts of interest to declare.

Re: Disparities in Utilization of Autologous Hematopoietic Cell Transplantation for Treatment of Multiple Myeloma

Costa et al. recently reported that racial disparities prevented nearly 40% of non-Hispanic blacks with multiple myeloma (MM) from undergoing stem cell transplantation (SCT), but the authors were unable to provide an explanation for the disparities because of limitations of their datasets. They hypothesized that socioeconomic status (SES) and/or insurance providers might account for the disparity. To examine the issue raised by Costa et al., we performed a secondary analysis using hierarchical multivariate logistic regression with data previously collected to determine if age at diagnosis, sex, SES, primary insurance provider at diagnosis, and comorbidity score help explain the racial disparities in SCT utilization. A model of race, age, sex, SES, insurance provider, and comorbidity score was the most accurate model in predicting stem cell utilization status (χ2[12] = 193.859; P < .001; area under the curve = .837; P < .001). After controlling for the covariates, black patients were less likely to undergo SCT than white patients (adjusted odds ratio, .49; 95% confidence interval, .27 to .89; P = .013). In conclusion, we also observed racial disparities between black and white patients with MM in SCT utilization and these are not fully accounted for by the covariates age, sex, SES, insurance provider, and comorbidity score.

Survival Outcomes Of Very Young (

BackgroundMultiple myeloma (MM) is uncommon in patients below 40 years of age. Between 1960 and 1992 when stem cell transplantation (SCT) was rarely performed and novel agents were unavailable, overall survival (OS) was reported to be 54 months in the Mayo Clinic series of patients with MM below forty years of age. The impact of novel agents or SCT in this population is unclear. Herein, we report the outcomes of patients under 40 years of age who were evaluated over a span of more than five decades with a focus on the periods contemporaneous with the introduction of SCT and novel agents, the major therapeutic milestones in the management of MM. MethodsWe analyzed the medical records of patients with MM under 40 years of age who were evaluated at Mayo Clinic, Rochester, MN between 1/1/1960 and 12/31/2011. SCT and novel agents were utilized infrequently prior to the years 1990 and 2000, respectively. Patients were therefore divided into three cohorts by the year of diagnosis of MM: Group A (1960 -1979), Group B (1980 to 1999) and Group C (2000 –2011) to assess the impact of major therapeutic advances (i.e. Increased utilization of SCT and novel agents compared to conventional chemotherapy). OS was calculated by the Kaplan -Meier method. ResultsOut of 8063 unique MM patients seen at our institution during this period, 174 (2%) patients were under 40 years of age at diagnosis. The median age at diagnosis was 36 years (range: 19-39). The estimated median follow-up of the entire cohort was 179 months (95% CI: 149-196 months). The median OS of the entire cohort was 76 months (95% CI: 45-80 months) from diagnosis. The median OS of patients in Groups A (n=29, 17%), B (n=93, 53%), and C (n=52, 30% ) was 44 months (95% CI: 20- 76), 79 months (95% CI: 46-97), and 92 months (95% CI: 42-NR), respectively (p=0.005). No significant OS difference was noted between Groups B and C (Figure). Sixty-six (38%) patients underwent at least one SCT (autologous: 56 patients and allogeneic 10 patients). The utilization of SCT increased over the three time periods from Group A 0%, Group B 39% and Group C 58% (p<0.001). Of 153 patients in whom front-line therapy-related data were available 118 (77%) received conventional and 35(23%) received novel agents. The median OS of patients who underwent transplantation (n=66) was 93 months compared to 46 months for those who did not (n=108, p=.01). In the 66 patients who underwent SCT, the median OS was 93 months for conventional agents-based induction (95% CI: 73-143) versus 97 months (95% CI: 41-187) for novel agent based induction (p=.4). [Display omitted] Sixty-eight (40%) patients received novel agents at some point during the course of their disease [bortezomib 35 (54%), thalidomide 44 (67%), lenalidomide 47 (71%) and pomalidomide 7(11%)]. The median survival of these patients was 131 (95% CI: 88-161) months versus to 46 (CI: 37-74) months for those (n=106) receiving conventional agents only (p=0.0002). ConclusionsOS of patients with MM under 40 years of age in the pre-transplant era (Group A) was significantly shorter than that of patients diagnosed in the era of SCT (Group B) and SCT plus novel agents (Group C). Although improvement of survival outcomes is evident with utilization of novel agents, similar outcomes of Groups B and C indicate lack of incremental benefit over SCT with utilization of novel agents. Maximal survival gain coincides with the introduction of SCT in this unique patient cohort. Disclosures:Kumar:Celgene: Consultancy, Research Funding; Millennium: Consultancy, Research Funding; Onyx: Consultancy, Research Funding.

Use of stem cell transplantation (SCT) as initial therapy in multiple myeloma (MM) and impact of sociodemographic factors in the era of novel agents: Analysis of 137,409 patients using the National Cancer Data Base (NCDB).

8580 Background: Novel agents for MM became available in the past decade. Our study aim was to determine whether the rate of SCT utilization as part of initial therapy in patients with MM had changed in the US after the introduction of novel agents. We also determined the impact of various sociodemographic factors. Methods: De-identified patient level data were obtained from the NCDB bParticipant User File. NCDB is a nationwide oncology outcomes database, which comprises approximately 70% of all newly diagnosed cancer cases in the United States. Results: A total of 137,409 MM patients were included in the study. The median age was 68 years (range, 18-90+). The population was predominantly male (54%), white (79%), non-Hispanic (87%), and living in a metro (78%), low- or middle-income (annual income &lt; $46,000; 63%), and low- or moderate-education (&gt; 14% without high school diploma; 66%) areas. Most patients had minimal or no co-morbidities (Charlson-Deyo score of 0-1; 93%) and treated in the community setting (62%). The rate of SCT use as initial therapy consistently increased annually from 5% in 1998 to 12% in 2010. This was true for all socio-demographic subgroups except for those who were age &gt; 75 years, treated in the Northeast, and the medically uninsured. Multivariate logistic regression showed that the following characteristics were significant main predictors of lower likelihood of SCT use (odds ratio): age &gt; 65 years (0.18), non-white race (0.57), Hispanic ethnicity (0.68), lack of high education (0.89), annual income &lt; $30,000 (0.76), diagnosed before the era of novel agents (1998-2003) (0.70), covered by a non-managed care insurance (0.57), treatment in a non-academic facility (0.28), residence at a non-metro area (0.88), and treatment facility located outside of the Midwest or West (0.62). In contrast, sex was not significant. Conclusions: Even after the introduction of novel agents, the rate of SCT as initial therapy in MM continues to increase annually in the US. Significant disparity exists in the rate of SCT use by demographic, social, and geographic factors as well as the type of treatment facility.

Stem Cell Transplantation for Fanconi Anemia: A Survey of Decision-Making among Families in the US and Canada.

Abstract 3207Poster Board III-144 IntroductionFanconi anemia (FA) is a primarily autosomal recessive, genetically heterogeneous disorder of chromosomal instability associated with congenital anomalies, cancer susceptibility, and progressive bone marrow failure. Currently, hematopoietic stem-cell transplantation (SCT) is the only treatment that will restore normal hematopoeisis; transplant protocols and recommendations are modified routinely due to challenges with severe toxicities in pre-transplantation conditioning regimens as well as complications related to acute and chronic graft-versus-host disease. SCT may also increase the risk of subsequent solid tumors, particularly head and neck squamous cell carcinomas, and has a significant mortality rate among FA patients. Given these potential harms, the decision-making process about SCT in FA patients is complex. The purpose of this study was to survey FA patients and their families in the United States (USA) and Canada to gain a better understanding of the factors that influence their decisions about SCT. We explored how perceptions of the need and associated risks of SCT influence the choice to undergo SCT. Patients and MethodsIndividuals who were members of the FA family organizations in the USA and Canada were sent a self-report survey via a two-phase blinded mailing whereby non-responders from the first mailing were sent a second questionnaire. Parents and patients were queried separately. A single respondent was selected to represent each affected individual, according to the following selection hierarchy: mother as primary respondent, followed by father, adult patient, and spouse/significant other. Survey items about decision-making were adapted from The Beliefs about Medicines Questionnaire (Horne, Weinman, & Hankins, 1999) which assesses respondents' beliefs about the necessity, associated risks, and concerns regarding a medical intervention, in this case SCT. Each of the three predictor variables (necessity, risk and concern) was measured by core items rated on a 4-point Likert scale. The necessity subscale was measured by core items which evaluated the respondents' perception of the medical need for SCT. The risk subscale measured respondents' perceived likelihood that the patient would suffer from: physical or emotional side-effects of SCT, liver disease, infection, graft-versus-host-disease, infertility, post-SCT cancer, post-SCT leukemia, graft rejection, death, or hazards to future offspring. The concern subscale consisted of respondents' evaluation of the degree to which they were concerned about: physical or emotional side-effects of SCT, short or long-term side-effects, death, uncertainty about the procedure, effect of SCT on the patient's ability to do daily activities, financial difficulties, finding an adequate donor, and conflicts with religious or ethical beliefs. Logistic regression was used to examine the direct effect of each of the three predictor variables on the outcome variable, selection or refusal of SCT. ResultsRespondents for 223 individuals with FA (44% of those surveyed) were included in this analysis. The median age of patients at the time of diagnosis was 4.8 years (0-41 years); the median age at the time of transplant was 9.1 years (1.7-41.2 years). The majority of respondents were mothers (85%); fathers represented 10% of the final sample. A majority of patients included in the present analysis had SCT (83%), and a smaller majority were living at the time the survey was completed (64%). The subscale items exhibited good internal consistency reliability (αa > 0.80). In bivariate analyses, only perceived necessity and risk showed significant relationships with the outcome variable, SCT (p < 0.01). However, in multivariate logistic regression analyses including all three predictor variables, only perceived necessity of SCT was significantly associated with the decision to undergo SCT. ConclusionsThis is the first study to explore factors related to the utilization of SCT in FA. Our results demonstrated that the majority of subjects opted for SCT and that perceived necessity and risk of SCT were significantly related to the decision, while concern related to the SCT procedure was not. The perceived necessity of SCT was the most important factor for decision-making. These results suggest areas of emphasis for future research and counseling of FA patients and their family members. DisclosuresNo relevant conflicts of interest to declare.