Plain language summary of the MAIA study of daratumumab plus lenalidomide and dexamethasone for the treatment of people with newly diagnosed multiple myeloma.

WHAT IS THIS SUMMARY ABOUT?: This is a summary of a clinical trial called MAIA. The trial tested 2 combinations of cancer drugs (daratumumab plus lenalidomide and dexamethasone compared with lenalidomide and dexamethasone) in people with newly diagnosed multiple myeloma. None of the participants who took part in the study had been treated before or were eligible to receive stem-cell transplants. HOW WAS THE STUDY IN THIS SUMMARY CONDUCTED?: A total of 737 participants took part. Half of the participants took daratumumab plus lenalidomide and dexamethasone, while the other half of the participants took only lenalidomide and dexamethasone. Once participants started taking the drugs, the cancer was monitored for improvement (response to treatment), worsening (disease progression), or no change. Participants' blood and urine were tested for myeloma protein to measure response to the treatment. Participants were also monitored for side effects. WHAT WERE THE RESULTS OF THE STUDY?: After approximately 56 months of follow-up, more participants who took daratumumab plus lenalidomide and dexamethasone were alive and had decreased myeloma protein levels (indicating improvement of cancer) than participants who took only lenalidomide and dexamethasone. The most common side effects were abnormally low white and red blood cell counts and increased lung infections. WHAT DO THE RESULTS OF THE STUDY MEAN?: In the MAIA study, participants with multiple myeloma who took daratumumab plus lenalidomide and dexamethasone lived longer and had decreased myeloma protein levels than participants who took only lenalidomide and dexamethasone, indicating survival could be more likely with daratumumab added. Clinical Trial Registration: NCT02252172 (Phase 3 MAIA study).

Impact of Disease Progression, Line of Therapy, and Response on Health-Related Quality of Life in Multiple Myeloma: A Systematic Literature Review.

This systematic literature review (SLR) was conducted to better understand the impact of disease progression, line of therapy, and clinical response on health-related quality of life (HRQoL) in patients with multiple myeloma (MM). Multiple databases were searched to identify records relating to HRQoL in adult patients with MM. Titles and abstracts were independently screened by 2 reviewers for inclusion based on pre-defined criteria. Records flagged for inclusion had full texts subsequently screened using the same method. A third round of screening was then conducted to identify studies that assessed the relationship of HRQoL to disease progression, line of therapy, or clinical response. Quality assessment was conducted on utility studies using the National Institute for Health and Care Excellence Quality Assessment Checklist for Health State Utility Values. After all rounds of screening were complete, 44 records (representing 41 studies) were included in the SLR. Thirty records reported data relating HRQoL to disease progression, 5 reported data relating HRQoL to line of therapy, and 19 reported data relating HRQoL to response. Despite a lack of homogeneity and small number of studies, the data show overall that progressive disease and increasing lines of therapy were associated with worsened patient HRQoL and increasing depth of response was associated with improved patient HRQoL. The findings from this SLR support that desirable treatment outcomes such as delayed progression, fewer lines of therapy, and achieving the deepest possible clinical response result in improved HRQoL in patients with MM.

Quality of Life and Pain Experienced by Children and Adolescents With Cancer at Home Following Discharge From the Hospital.

An exploratory study was conducted to examine the quality of life and pain experienced by patients with pediatric cancer at home after discharge. Physical, cognitive, social, and emotional aspects of quality of life were measured and how these may be affected by age, sex, diagnosis, and pain status. The authors also characterized intensity, location, and quality of pain experienced. A sample of 33 patients participating in a larger study was selected on the basis of having pain on the day of discharge and having completed the Pediatric Quality of Life Inventory Generic, Cancer Module, Multidimensional Fatigue Scale, and the Adolescent Pediatric Pain Tool at home. Cancer diagnoses were leukemias/lymphomas (42.4%), brain/central nervous system tumors (27.3%), sarcomas (24.2%), or other (6.1%). More than half of patients reported pain (n=17; 51.5%). Patients with pain had more fatigue affecting the quality of life (P=0.01), and lower physical and emotional functioning, leading to lower overall health-related quality of life scores (P=0.011). Female individuals and adolescents reported worse emotional functioning (P=0.02 and P=0.05, respectively). Physical, cognitive, and social functioning were lowest among patients diagnosed with sarcomas (P=0.00, P=0.01, and P=0.04, respectively). It is important to understand the symptom experience of patients at home as a first step in moving towards optimal discharge teaching and treatment.

Use of a Pediatric Bleeding Questionnaire in the Screening of Von Willebrand Disease in Young Females at Menarche in the Primary Care Setting.

Von Willebrand disease (VWD), the most common inherited bleeding disorder, is caused by deficiency or dysfunction in von Willebrand factor. Assessment of hemorrhagic symptoms is essential for early diagnosis, although bleeding histories are taken in a nonstandardized manner. Validated bleeding assessment tools provide objectivity in evaluating bleeding patterns of females at menarche and may improve provider confidence in screening for VWD. Utilizing a pretest/posttest design, in this project we implemented and evaluated the use of a pediatric bleeding questionnaire in eight pediatric primary care clinics for 3 months. Results indicate improved provider knowledge, confidence, and skills after implementation. The importance and quality of the tool were rated highly by the providers, while the ease of use was rated low. Providers were satisfied with the practice change and believed that it improved their clinical abilities. The use of this tool can improve VWD screening in this practice setting.

Effects of Leukoreduction and Premedication With Acetaminophen and Diphenhydramine in Minimizing Febrile Nonhemolytic Transfusion Reactions and Allergic Transfusion Reactions During and After Blood Product Administration: A Literature Review With Recommendations for Practice.

Transfusion-related reactions cause unwanted interruptions in blood-product administration and potential complications for patients. The most common reactions are febrile nonhemolytic transfusion reactions (FNHTRs) and allergic transfusion reactions (ATRs). The presence of leukocytes in blood products has been associated with these reactions, and efficacy of leukoreduction in minimizing FNHTRs and ATRs has recently been investigated. In addition, premedication with acetaminophen and diphenhydramine is the most widely used practice in minimizing FNHTRs and ATRs, yet the benefit of this is not supported by research. The aim of this systematic literature review was to evaluate the potential benefits of both of these interventions in minimizing FNHTRs and ATRs and provide recommendations for practice. We found moderate quality evidence with strong recommendations for the practice of leukoreduction in minimizing FNHTRs but not ATRs. We did not find evidence to support the use of premedications in minimizing transfusion-related reactions, and we question the need for this practice in settings where leukoreduction is used.

Remote monitoring of pain and symptoms using wireless technology in children and adolescents with sickle cell disease.

PURPOSE: The purpose of this study was to examine (a) symptoms, (b) pain characteristics (intensity, location, quality), (c) pain medications and nonpharmacological strategies used for pain, (d) thoughts and feelings, and (e) healthcare visits. We also examined the relationship between pain and sleep. DATA SOURCES: Pain and symptoms were entered on an electronic e-Diary using a smartphone and were remotely monitored by an advanced practice registered nurse (APRN). Sixty-seven children and adolescents (10-17 years) reported mild to severe pain at home that did not require healthcare visits. Symptoms reported were (a) general symptoms such as tiredness/fatigue (34.7%), headache (20.8%), yellowing of the eyes (28.4%); (b) respiratory symptoms such as sniffling (32.9%), coughing (19.1%), changes in breathing (10.0%); and (c) musculoskeletal symptoms such as stiffness in joints (15.8%). A significant negative correlation was found between pain and sleep (r = -.387, p = .024). Factors that predict pain included previous history of sickle cell disease (SCD) related events, symptoms, and negative thoughts. CONCLUSION: Pain and multiple symptoms entered on a web-based e-Diary were remotely monitored by an APRN and prompted communications, further evaluation, and recommendations. IMPLICATIONS FOR PRACTICE: Remote monitoring using wireless technology may facilitate timely management of pain and symptoms and minimize negative consequences in SCD.

Facilitating pediatric patient-provider communications using wireless technology in children and adolescents with sickle cell disease.

INTRODUCTION: Use of wireless devices has the potential to transform delivery of primary care services for persons with sickle cell disease (SCD). The study examined text message communications between patients and an advanced practice registered nurse (APRN) and the different primary care activities that emerged with use of wireless technology. METHODS: Patients (N = 37; mean age 13.9 ± 1.8 years; 45.9% male and 54.1% female) engaged in intermittent text conversations with the APRN as part of the Wireless Pain Intervention Program. Content analyses were used to analyze the content of text message exchanges between patients and the APRN. RESULTS: The primary care needs that emerged were related to pain and symptom management and sickle cell crisis prevention. Two primary care categories (collaborating and coaching), four primary care subcategories (screening, referring, informing, and supporting), and 16 primary care activities were evident in text conversations. DISCUSSION: The use of wireless technology may facilitate screening, prompt management of pain and symptoms, prevention or reduction of SCD-related complications, more efficient referral for treatments, timely patient education, and psychosocial support in children and adolescents with SCD.

Usability testing of a Smartphone for accessing a web-based e-diary for self-monitoring of pain and symptoms in sickle cell disease.

We examined the usability of smartphones for accessing a web-based e-Diary for self-monitoring symptoms in children and adolescents with sickle cell disease (SCD). One group of participants (n = 10; mean age, 13.1 ± 2.4 y; 5 M; 5 F) responded to questions using precompleted paper-based measures. A second group (n = 21; mean age, 13.4 ± 2.4 y; 10 M; 11 F) responded based on pain and symptoms they experienced over the previous 12 hours. The e-Diary was completed with at least 80% accuracy when compared to paper-based measures. Symptoms experienced over the previous 12 hours included feeling tired (33.3%), headache (28.6%), coughing (23.8%), lack of energy/fatigue (19.0%), yellowing of the eyes (19.0%), pallor (19.0%), irritability (19.0%), stiffness in joints (19.0%), general weakness (14.3%), and pain (14.3%), rating on average as 2.0 ± 1.7 (on 0 to 10 scale). Overall, sleep was good (8.1 ± 1.4 on the 0 to 10 scale). In conclusion, children with SCD were able to use smartphones to access a web-based e-Diary for reporting pain and symptoms. Smartphones may improve self-reporting of symptoms and communication between patients and their health care providers, who may consequently be able to improve pain and symptom management in children and adolescents with SCD in a timely manner.