scholarly journals Development and Pilot Testing of Text Messages to Help Reduce Sugar-Sweetened Beverage Intake Among Rural Caregivers and Adolescents: Mixed Methods Study

10.2196/14785 ◽  
2019 ◽  
Vol 7 (7) ◽  
pp. e14785
Author(s):  
Maryam Yuhas ◽  
Kathleen J Porter ◽  
Donna-Jean P Brock ◽  
Annie Loyd ◽  
Brittany A McCormick ◽  
...  
Keyword(s):  
Mixed Methods ◽  
Focus Groups ◽  
Phase 1 ◽  
Pilot Trial ◽  
Text Message ◽  
Text Messages ◽  
Phase 2 ◽  
Rural Caregivers ◽  
Caregiver Behaviors

Background A high consumption of sugar-sweetened beverages (SSBs) poses significant health concerns, particularly for rural adults and adolescents. A manner in which the health of both caregivers and adolescents can be improved is by developing innovative strategies that target caregivers as the agents of change. Sending text messages through mobile phones has been cited as an effective way to improve behavioral outcomes, although little research has been conducted in rural areas, particularly focusing on SSB intake. Objective By targeting rural caregivers, this 2-phase study aimed to (1) understand caregivers’ perceptions and language preferences for SSB-related text messages to inform and refine message development and delivery and (2) evaluate the acceptability of text messages for SSB intake behavior change and examine short-term effects on SSB intake behavior. Methods A convergent mixed methods design was used to systematically develop and pilot-test text messages with caregivers in Southwest Virginia. In phase 1, 5 focus groups that included a card-sorting activity were conducted to explore advantages/disadvantages, language preferences (ie, tone of voice, audience, and phrase preferences), and perceived use of text messages. In phase 2, caregivers participated in a 5-week text message pilot trial that included weekly educational and personalized strategy messages and SSB intake assessments at baseline and follow-up. Before the focus groups and after completing the pilot trial, caregivers also completed a pre-post survey that assessed SSB intake, SSB home availability, and caregivers’ SSB-related practices. Caregivers also completed individual follow-up telephone interviews following the pilot trial. Results In phase 1, caregivers (N=33) reported that text messages were convenient, accessible, and easy to read. In addition, they preferred messages with empathetic and authoritative tones that provided useful strategies and stayed away from using absolute words (eg, always and never). In the phase 2 pilot trial (N=30), 87% of caregivers completed baseline and 77% completed follow-up assessment, suggesting a high utilization rate. Other ways in which caregivers reported benefiting from the text messages included sharing messages with family members and friends (80%), making mental notes (57%), and looking back at messages as reminders (50%). Caregivers reported significant improvements in home environment, parenting practices, and rulemaking around SSB (P=.003, P=.02, and P=.04, respectively). In addition, the frequency of SSB intake among caregivers and adolescents significantly decreased (P=.003 and P=.005, respectively). Conclusions Spending time in the formative phases of text message development helped understand the unique perspectives and language preferences of the target population. Furthermore, delivering an intervention through text messages has the potential to improve caregiver behaviors and reduce SSB intake among rural caregivers and adolescents. Findings from this study were used to develop a larger bank of text messages, which would be used in a future study, testing the effectiveness of a text message intervention targeting SSB intake–related caregiver behaviors.

scholarly journals Development and Pilot Testing of Text Messages to Reduce Sugar-Sweetened Beverage Intake in Rural Caregivers and Adolescents: A Mixed-Methods Study (Preprint)

2019 ◽  
Author(s):  
Maryam Yuhas ◽  
Kathleen Joyce Porter ◽  
Donna-Jean Brock ◽  
Annie Loyd ◽  
Brittany Alexandra McCormick ◽  
...  
Keyword(s):  
Mixed Methods ◽  
Focus Groups ◽  
Pilot Trial ◽  
Text Message ◽  
Text Messages ◽  
Utilization Rate ◽  
Rural Caregivers ◽  
Message Development ◽  
Caregiver Behaviors

BACKGROUND High consumption of sugar-sweetened beverages (SSB) poses significant health concerns, particularly for rural adults and adolescents. Developing innovative strategies that target caregivers as the agents of change could be a promising way to improve both caregiver and adolescent health. Sending text messages through mobile phones has been cited as an effective way to improve behavioral outcomes, although little research has been conducted in rural areas, particularly focusing on SSB intake. OBJECTIVE Targeting rural caregivers, the objectives of this two phase study were to: 1) understand caregivers’ perceptions and language preferences for SSB-related text messages to inform and refine message development and delivery, and 2) evaluate the acceptability of text messages for SSB behavior change, and examine short-term effects on SSB behaviors. METHODS A convergent mixed-methods design was used to systematically develop and pilot test text messages with caregivers in Southwest Virginia. In phase one, five focus groups that included a card-sorting activity were conducted to explore advantages/disadvantages, language preferences (i.e., tone of voice, audience, phrase preferences), and perceived use of text messages. In phase two, caregivers participated in a 5-week text message pilot trial that included weekly educational and personalized strategy messages and SSB assessments at baseline and follow-up. Before the focus groups and after completing the pilot trial, caregivers also completed a pre-post survey that assessed SSB intake, SSB home availability, and caregiver SSB-related practices. Caregivers also completed individual follow-up telephone interviews following the pilot trial. RESULTS In phase one, caregivers (n=33) reported that text messages were convenient, accessible, and easy to read. Additionally, caregivers preferred messages with empathetic and authoritative tones of voice that provided useful strategies and stayed away from using absolute words (e.g., always, never). In the phase two pilot trial (n=30), 87% of caregivers completed baseline and 77% completed follow-up assessment suggesting a high utilization rate. Other ways caregivers reported benefiting from the text messages included sharing messages with family members, friends, and coworkers (80%), making mental notes (57%), and looking back at messages as reminders (50%). Caregivers reported significant improvements in home environment, parenting practices, and rulemaking around SSB (all P <0.05). Also, the frequency of SSB intake among caregivers and adolescents significantly decreased (P <0.01). CONCLUSIONS Spending time in the formative phases of text message development helped to understand the unique perspectives and language preferences of the target population. This study also found that delivering an intervention through text messages has the potential to improve caregiver behaviors and reduce SSB intake in rural caregivers and adolescents. Findings from this study were used to develop a larger bank of text messages, which will be used in a future study testing the effectiveness of a text message intervention targeting SSB-related caregiver behaviors.

A text-message intervention for adolescents with depression and their parent/caregiver to overcome cognitive barriers to mental health treatment initiation: Focus groups and a pilot trial (Preprint)

2021 ◽  
Author(s):  
Brian Suffoletto ◽  
Tina Goldstein ◽  
David Brent
Keyword(s):  
Mental Health ◽  
Focus Groups ◽  
Mental Health Treatment ◽  
Phase 1 ◽  
Pilot Trial ◽  
Text Message ◽  
Phase 2 ◽  
Test Engagement ◽  
Text Message Intervention

BACKGROUND Many adolescents with depression do not pursue mental health (MH) treatment following referral by a heathcare provider. We developed a theory-based automated text message intervention (Text to Connect, T2C) that attempts to reduce cognitive barriers to initiation of MH care. OBJECTIVE In this 2-phase study, we sought to first understand potential for T2C and then test engagement, usability, and potential efficacy of T2C among adolescents with depression and their parent/caregiver. METHODS In phase 1, we conducted focus groups with adolescents with depression (n=9) and their parent/caregiver (n=9) separately and transcripts were examined for themes. In phase 2, we conducted an open trial of T2C with adolescents with depression referred to MH care (n=43) and their parent/caregiver (n=28). We assessed usability by examining program engagement, usability ratings and qualitative feedback at 4-weeks follow-up. We assessed potential effectiveness by examining change in perceived barriers to treatment and MH care initiation from baseline to 4-weeks. RESULTS In phase 1, we found that themes supported the T2C approach. In phase 2, we found high engagement with daily negative affect check-ins, high usability ratings, and self-reported decreases in barriers to MH treatment over time among adolescents. At 4-weeks follow-up, most adolescents reported either having initiated MH care (52%) or pending scheduling (15%), leaving only 15% of adolescents still not interested in MH care. CONCLUSIONS Findings from this study suggest that T2C is acceptable to adolescents with depression and most parent/caregivers, is used at high rates, and may be helpful to reduce cognitive barriers to MH care initiation. CLINICALTRIAL NCT04560075

scholarly journals Usability and Acceptability of a Text Message-Based Developmental Screening Tool for Young Children: Pilot Study (Preprint)

2018 ◽  
Author(s):  
Pamela Ryden Johnson ◽  
Jessica Bushar ◽  
Margaret Dunkle ◽  
Sharon Leyden ◽  
Elizabeth T Jordan
Keyword(s):  
Focus Groups ◽  
Low Income ◽  
Screening Tool ◽  
Text Message ◽  
Text Messages ◽  
Developmental Screening ◽  
Screening Questions ◽  
Low Income Mothers ◽  
Developmental Screening Tool

BACKGROUND Only 30% of parents of children aged 9-35 months report that their child received a developmental screening in the previous year. Screening rates are even lower in low-income households, where the rates of developmental delays are typically higher than those in high-income households. Seeking to evaluate ways to increase developmental screening, Text4baby, a national perinatal texting program, created an interactive text message-based version of a validated developmental screening tool for parents. OBJECTIVE This study aimed to assess whether a text message-based developmental screening tool is usable and acceptable by low-income mothers. METHODS Low-income mothers of infants aged 8-10 months were recruited from the Women, Infants and Children Program clinics in Prince George’s County, MD. Once enrolled, participants used text messages to receive and respond to six developmental screening questions from the Parents’ Evaluation of Developmental Status: Developmental Milestones. After confirming their responses, participants received the results and feedback. Project staff conducted a follow-up phone survey and invited a subset of survey respondents to attend focus groups. A representative of the County’s Infants and Toddlers Program met with or called participants whose results indicated that their infants “may be behind.” RESULTS Eighty-one low-income mothers enrolled in the study, 93% of whom reported that their infants received Medicaid (75/81). In addition, 49% of the mothers were Hispanic/Latina (40/81) and 42% were African American (34/81). A total of 80% participated in follow-up surveys (65/81), and 14 mothers attended focus groups. All participants initiated the screening and responded to all six screening questions. Of the total, 79% immediately confirmed their responses (64/81), and 21% made one or more changes (17/81). Based on the final responses, 63% of participants received a text that the baby was “doing well” in all six developmental domains (51/81); furthermore, 37% received texts listing domains where their baby was “doing well” and one or more domains where their baby “may be behind” (30/81). All participants received a text with resources for follow-up. In a follow-up survey reaching 65 participants, all respondents said that they would like to answer screening questions again when their baby was older. All but one participant would recommend the tool to a friend and rated the experience of answering questions and receiving feedback by text as “very good” or “good.” CONCLUSIONS A mobile text version of a validated developmental screening tool was both usable and acceptable by low-income mothers, including those whose infants “may be behind.” Our results may inform further research on the use of the tool at older ages and options for a scalable, text-based developmental screening tool such as that in Text4baby.

scholarly journals MyDiaText™: Feasibililty and Functionality of a Text Messaging System for Youth With Type 1 Diabetes

2019 ◽  
Vol 45 (3) ◽  
pp. 253-259 ◽  
Author(s):  
Tara Kaushal ◽  
Kathleen A. Montgomery ◽  
Reid Simon ◽  
Kelly Lord ◽  
Jennifer Dougherty ◽  
...  
Keyword(s):  
Type 1 Diabetes ◽  
User Satisfaction ◽  
Text Messaging ◽  
Pilot Trial ◽  
Text Message ◽  
Text Messages ◽  
Support Tool ◽  
Reading Text

Purpose The purpose of this study was to determine the feasibility and functionality of MyDiaText™, a website and text messaging platform created to support behavior change in adolescents with type 1 diabetes (T1DM) and to evaluate user satisfaction of the application. Methods This study was a nonrandomized, prospective, pilot trial to test the feasibility and user interface with MyDiaText, a text message system for 10- to 17-year-old youths with newly diagnosed T1DM. Feasibility was evaluated by assessing for the user’s ability to create a profile on the website. Functionality was defined by assessing whether a subject responded to at least 2 text messages per week and by their accumulating points on the website. User satisfaction of the text messaging system was assessed using an electronic survey. The 4 phases of this study were community engagement—advisory sessions, screening and enrollment, intervention, and follow-up. Results Twenty subjects (14 male, 6 female) were enrolled. All subjects were able to create a profile, and of these, 86% responded to at least 2 text messages per week. A survey administered during follow-up showed that users enjoyed reading text messages, found them useful, and thought the frequency of messages was appropriate. Conclusion MyDiaText is a feasible, functional behavioral support tool for youth with T1DM. Users of the application reported high satisfaction with text messages and the reward system.

Understanding Diabetes-Related Distress Characteristics and Psychosocial Support Preferences of Urban African American Adults Living With Type 2 Diabetes: A Mixed-Methods Study

2018 ◽  
Vol 44 (2) ◽  
pp. 144-157 ◽  
Author(s):  
Sula Hood ◽  
Amy Irby-Shasanmi ◽  
Mary de Groot ◽  
Elissabeth Martin ◽  
Andrew S. LaJoie
Keyword(s):  
Type 2 Diabetes ◽  
African American ◽  
Mixed Methods ◽  
Focus Groups ◽  
Psychosocial Support ◽  
Phase 1 ◽  
Culturally Appropriate ◽  
African American Adults

Purpose The purpose of this study is to understand diabetes-related distress (DRD) characteristics and identify psychosocial support preferences of urban African American adults living with type 2 diabetes (T2DM). Methods A 2-phase, mixed-methods sequential explanatory study design was used to gather data. In phase 1, a purposive sample of participants (N = 155) was recruited and asked to complete a written survey. The Diabetes Distress Scale (DDS17) was used to assess DRD, including subscales—emotional burden (EB), regimen distress (RD), interpersonal distress (ID), and physician distress (PD). In phase 2, a subset of phase 1 participants (N = 23) volunteered to attend 1 of 4 gender-stratified follow-up focus groups to contextualize the quantitative survey results. Results Survey findings indicate that on average, participants had moderate levels of DRD (aggregate), RD, and EB but had low ID and PD. During follow-up focus groups, participants described RD and EB as their primary distress types and emphasized that clinicians should prioritize the mental health aspects of T2DM similarly to its physical aspects. Participants expressed a desire for culturally appropriate peer support groups as a psychosocial support resource for distress coping and specifically requested the development of gender-stratified groups and groups for young adults. Conclusions Results support the need to screen for and address diabetes-related distress among African American patients with T2DM. Findings also inform the development of culturally appropriate psychosocial support resources to facilitate diabetes-related distress coping.

A Pilot Study of Mobile Telephone Message Interventions with Suicide Attempters in China

Crisis ◽  
2010 ◽  
Vol 31 (2) ◽  
pp. 109-112 ◽  
Author(s):  
Hui Chen ◽  
Brian L. Mishara ◽  
Xiao Xian Liu
Keyword(s):  
Pilot Study ◽  
Effective Means ◽  
Text Message ◽  
Text Messages ◽  
Mobile Telephone ◽  
Future Research ◽  
Quick Method ◽  
Suicide Attempters ◽  
To Receive

Background: In China, where follow-up with hospitalized attempters is generally lacking, there is a great need for inexpensive and effective means of maintaining contact and decreasing recidivism. Aims: Our objective was to test whether mobile telephone message contacts after discharge would be feasible and acceptable to suicide attempters in China. Methods: Fifteen participants were recruited from suicide attempters seen in the Emergency Department in Wuhan, China, to participate in a pilot study to receive mobile telephone messages after discharge. All participants have access to a mobile telephone, and there is no charge for the user to receive text messages. Results: Most participants (12) considered the text message contacts an acceptable and useful form of help and would like to continue to receive them for a longer period of time. Conclusions: This suggests that, as a low-cost and quick method of intervention in areas where more intensive follow-up is not practical or available, telephone messages contacts are accessible, feasible, and acceptable to suicide attempters. We hope that this will inspire future research on regular and long-term message interventions to prevent recidivism in suicide attempters.

scholarly journals Quantitative Checklist for Autism in Toddlers (Q-CHAT). A population screening study with follow-up: the case for multiple time-point screening for autism

2021 ◽  
Vol 5 (1) ◽  
pp. e000700
Author(s):  
Carrie Allison ◽  
Fiona E Matthews ◽  
Liliana Ruta ◽  
Greg Pasco ◽  
Renee Soufer ◽  
...  
Keyword(s):  
Phase 1 ◽  
Autism Spectrum ◽  
Population Screening ◽  
Diagnostic Assessment ◽  
Test Accuracy ◽  
Multiple Time ◽  
Phase 2 ◽  
Time Points ◽  
Screening Study

ObjectiveThis is a prospective population screening study for autism in toddlers aged 18–30 months old using the Quantitative Checklist for Autism in Toddlers (Q-CHAT), with follow-up at age 4.DesignObservational study.SettingLuton, Bedfordshire and Cambridgeshire in the UK.Participants13 070 toddlers registered on the Child Health Surveillance Database between March 2008 and April 2009, with follow-up at age 4; 3770 (29%) were screened for autism at 18–30 months using the Q-CHAT and the Childhood Autism Spectrum Test (CAST) at follow-up at age 4.InterventionsA stratified sample across the Q-CHAT score distribution was invited for diagnostic assessment (phase 1). The 4-year follow-up included the CAST and the Checklist for Referral (CFR). All with CAST ≥15, phase 1 diagnostic assessment or with developmental concerns on the CFR were invited for diagnostic assessment (phase 2). Standardised diagnostic assessment at both time-points was conducted to establish the test accuracy of the Q-CHAT.Main outcome measuresConsensus diagnostic outcome at phase 1 and phase 2.ResultsAt phase 1, 3770 Q-CHATs were returned (29% response) and 121 undertook diagnostic assessment, of whom 11 met the criteria for autism. All 11 screened positive on the Q-CHAT. The positive predictive value (PPV) at a cut-point of 39 was 17% (95% CI 8% to 31%). At phase 2, 2005 of 3472 CASTs and CFRs were returned (58% response). 159 underwent diagnostic assessment, including 82 assessed in phase 1. All children meeting the criteria for autism identified via the Q-CHAT at phase 1 also met the criteria at phase 2. The PPV was 28% (95% CI 15% to 46%) after phase 1 and phase 2.ConclusionsThe Q-CHAT can be used at 18–30 months to identify autism and enable accelerated referral for diagnostic assessment. The low PPV suggests that for every true positive there would, however, be ~4–5 false positives. At follow-up, new cases were identified, illustrating the need for continued surveillance and rescreening at multiple time-points using developmentally sensitive instruments. Not all children who later receive a diagnosis of autism are detectable during the toddler period.

scholarly journals MP09: Canadian Community Utilization of Stroke Prevention Pilot Study-Emergency Department (C-CUSP ED)

CJEM ◽  
2017 ◽  
Vol 19 (S1) ◽  
pp. S68 ◽  
Author(s):  
R. Parkash ◽  
K. Magee ◽  
M. McMullen ◽  
M.B. Clory ◽  
M. D’Astous ◽  
...  
Keyword(s):  
Stroke Prevention ◽  
Phase 1 ◽  
Retrospective Chart Review ◽  
Phase 2 ◽  
Phase 3 ◽  
Three Phase ◽  
Rigorous Study ◽  
The Mean ◽  
Prior Risk

Introduction: Atrial fibrillation (AF) is the most common sustained arrhythmia affecting 1-2% of the population. Oral anticoagulation (OAC) reduces stroke risk by 60-80% in AF patients, but only 50% of indicated patients receive OAC. Many patients present to the ED with AF due to arrhythmia symptoms, however; lack of OAC prescription in the ED has been identified as a significant gap in the care of AF patients. Methods: This was a multi-center, pragmatic, three-phase before-after study, in three Canadian sites. Patients who presented to the ED with electrocardiographically (ECG) documented, nonvalvular AF and were discharged home were included. Phase 1 was a retrospective chart review to determine OAC prescription of AF patients in each ED; Phase 2 was a low-intensity knowledge translation intervention where a simple OAC-prescription tool for ED physicians with subsequent short-term OAC prescription was used, as well as an AF patient education package and a letter to family physicians; phase 3 incorporated Phase 2 interventions, but added immediate follow-up in a community AF clinic. The primary outcome of the study was the rate of new OAC prescriptions at ED discharge in AF patients who were OAC eligible and were not on OAC at presentation. Results: A total of 632 patients were included from June, 2015-November, 2016. ED census ranged from 30000-68000 annual visits. Mean age was 71±15, 67±12, 67±13 years, respectively. 47.5% were women, most responsible ED diagnosis was AF in 75.8%. The mean CHA2DS2-VASc score was 2.6±1.8, with no difference amongst groups. There were 266 patients eligible for OAC and were not on this at presentation. In this group, the prescription of new OAC was 15.8% in Phase 1 as compared to 54% and 47%, in Phases 2 and 3, respectively. After adjustment for center, components of the CHA2DS2-VASc score, prior risk of bleeding and most responsible ED diagnosis, the odds ratio for new OAC prescription was 8.0 (95%CI (3.5,18.3) p&lt;0.001) for Phase 3 vs 1, and 10.0 (95%CI (4.4,22.9) p&lt;0.001), for Phase 2 vs 1). No difference in OAC prescription was seen between Phases 2 and 3. Conclusion: Use of a simple OAC-prescription tool was associated with an increase in new OAC prescription in the ED for eligible patients with AF. Further testing in a rigorous study design to assess the effect of this practice on stroke prevention in the AF patients who present to the ED is indicated.

scholarly journals Codeveloping a multibehavioural mobile phone app to enhance social and emotional well-being and reduce health risks among Aboriginal and Torres Strait Islander women during preconception and pregnancy: a three-phased mixed-methods study

BMJ Open ◽  
2021 ◽  
Vol 11 (11) ◽  
pp. e052545
Author(s):  
Michelle Kennedy ◽  
Ratika Kumar ◽  
Nicole M Ryan ◽  
Jessica Bennett ◽  
Gina La Hera Fuentes ◽  
...  
Keyword(s):  
Mixed Methods ◽  
Mobile Phone ◽  
Phase 1 ◽  
Qualitative Interviews ◽  
Mixed Methods Study ◽  
Phase 2 ◽  
Community Members ◽  
Phase 3 ◽  
Aboriginal Community ◽  
Aboriginal Women

ObjectiveDescribe the development and pretest of a prototype multibehavioural change app MAMA-EMPOWER.DesignMixed-methods study reporting three phases: (1) contextual enquiry included stakeholder engagement and qualitative interviews with Aboriginal women, (2) value specification included user-workshop with an Aboriginal researcher, community members and experts, (3) codesign with Aboriginal researchers and community members, followed by a pretest of the app with Aboriginal women, and feedback from qualitative interviews and the user-Mobile Application Rating Scale (U-MARS) survey tool.SettingsAboriginal women and communities in urban and regional New South Wales, Australia.ParticipantsPhase 1: interviews, 8 Aboriginal women. Phase 2: workshop, 6 Aboriginal women. Phase 3: app trial, 16 Aboriginal women. U-MARS, 5 Aboriginal women.ResultsPhase 1 interviews revealed three themes: current app use, desired app characteristics and implementation. Phase 2 workshop provided guidance for the user experience. Phase 3 app trial assessed all content areas. The highest ratings were for information (mean score of 3.80 out of 5, SD=0.77) and aesthetics (mean score of 3.87 with SD of 0.74), while functionality, engagement and subjective quality had lower scores. Qualitative interviews revealed the acceptability of the app, however, functionality was problematic.ConclusionsDeveloping a mobile phone app, particularly in an Aboriginal community setting, requires extensive consultation, negotiation and design work. Using a strong theoretical foundation of behavioural change technique’s coupled with the consultative approach has added rigour to this process. Using phone apps to implement behavioural interventions in Aboriginal community settings remains a new area for investigation. In the next iteration of the app, we aim to find better ways to personalise the content to women’s needs, then ensure full functionality before conducting a larger trial. We predict the process of development will be of interest to other health researchers and practitioners.

scholarly journals A Phase 1/2 Trial of Pomalidomide, Dexamethasone and Pegylated Liposomal Doxorubicin for Patients with Relapsed/Refractory Multiple Myeloma

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 4774-4774
Author(s):  
James R. Berenson ◽  
Laura V. Stampleman ◽  
Alberto Bessudo ◽  
Peter J. Rosen ◽  
Leonard M Klein ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Progressive Disease ◽  
Liposomal Doxorubicin ◽  
Phase 1 ◽  
Single Agent ◽  
Pegylated Liposomal Doxorubicin ◽  
Great Promise ◽  
Phase 2 ◽  
Grade 3

Abstract Background Immunomodulatory drugs (IMiD), such as thalidomide and lenalidomide (LEN) and its newest derivative pomalidomide (POM), have shown great promise for the treatment of multiple myeloma (MM) patients (pts). POM has in vitro anti-MM potency and has shown efficacy for the treatment of relapsed/refractory (RR) MM pts. POM with dexamethasone (DEX) induces responses even for MM pts who are refractory to bortezomib (BORT) and LEN (Richardson et al, 2012). Pegylated liposomal doxorubicin (PLD) with BORT is FDA-approved for the treatment of MM pts who have received one prior therapy not containing BORT. The combination of PLD and LEN or thalidomide has shown efficacy for both RR and frontline MM pts (Offidani et al, 2006; 2007). We have also demonstrated that both the efficacy and tolerability of LEN in combination with DEX, PLD and BORT (DVD-R) may be improved by changing the doses and schedules of these drugs (Berenson et al, 2012). Based on these results, we hypothesized that the combination of POM, DEX and PLD would be effective for the treatment of RRMM pts. Thus, we conducted the first study investigating the safety and efficacy of POM in combination with intravenous (IV) DEX and PLD as a phase 1/2 trial using a modified dose, schedule and longer 28-day cycles for pts with RRMM. Methods The phase 1 portion enrolled MM pts w/ progressive disease whereas those enrolled in phase 2 also had to be refractory to LEN (single-agent or in combination), as demonstrated by progressive disease while receiving their last LEN-containing regimen or relapsed within 8 weeks of their last dose of this IMiD. Pts who have previously received POM treatment were ineligible. In the phase 1 portion, POM was administered at 2, 3 or 4 mg daily in three cohorts on days 1-21 of a 28-day cycle and DEX (40 mg) and PLD (5 mg/m2) were fixed and given intravenously on days 1, 4, 8, and 11. Results As of June 20th, 2014, 48 pts were enrolled in the trial and a total of 47 pts had received study drug. Pts had received a median of 4 prior treatments (range 1-18), with a median of 2 prior IMiD-containing regimens (range, 0-8). Fifty-three percent of the pts had received a prior PLD-containing regimen and 21% had received a prior IMiD and PLD combination treatment. Among all enrolled pts, 40 pts discontinued treatment and seven remain active. Pts completed a median of 3 cycles (range: 1-8), with a median follow-up time of 5.4 months (range: 0-22). During the phase 1 portion of the trial, the maximum tolerated dose (MTD) of POM was established at 4 mg. Enrollment of pts into the phase 2 portion of the trial began at the MTD. However, neutropenia ≥ grade 3 was observed at this dose in 10/17 (58.8%) phase 2 pts; and, as a result, the protocol was amended so that the MTD was lowered to 3 mg for all pts subsequently enrolled. Among the 36 pts enrolled in phase 2, 78% percent were refractory to LEN and steroids with or without other agents and 47% had previously received PLD. A median of 2 cycles (range, 1 to 8) were administered among the pts enrolled in phase 2. Thirty-five pts were evaluable for response as one pt was active but had not yet had any post-baseline disease assessments. Among all pts enrolled in phase 2, the overall response rate (ORR) and clinical benefit rate (CBR) were 29% and 49%, respectively, with 6 pts (17%) showing stable disease and 12 pts (34%) demonstrating progressive disease. For all pts enrolled in phase 2, the median follow-up time was 4.7 months (range 0-12) and the median PFS was 5.3 months. ORR and CBR for pts in the phase 2 were higher among pts receiving POM at 3 mg (32% and 58%, respectively) than among pts receiving POM at 4 mg (25% and 37%, respectively). Pts receiving the 4 mg dose of POM experienced more toxicities resulting in discontinuations, which likely explains the lower ORR and CBR observed among pts receiving this POM dose. Common ≥ grade 3 adverse events observed throughout the trial were neutropenia (21 pts; 44.7%), lymphopenia (10 pts; 21.3%), and hyponatremia (4 pts; 8.5%). One pt died of grade 5 sepsis. Conclusions This phase 1/2 trial is the first evaluating POM with PLD and DEX and demonstrates that the combination of POM at 3 mg, PLD and DEX using a modified 28-day cycle schedule is safe and effective for the treatment of MM pts refractory to LEN. Disclosures Berenson: Celgene: Consultancy, Honoraria, Research Funding, Speakers Bureau. Swift:Celgene: Consultancy, Honoraria. Vescio:Celgene: Honoraria.

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