Samara Rosenfeld

Early administration of rivipansel in patients with vaso-occlusive crises (VOCs) results in clinically meaningful benefits for adults and children with 

In fact, according to study findings presented at the 

62nd Annual American Society of Hematology (ASH) Meeting and Exposition

, early initiation of such treatment led to the shortening of IV opioid use and hospital stay. The findings suggested early initiation should be used to shorten or interrupt acute VOCs, similar to thrombolysis for heart attack or stroke.

Carlton Dampier, MD, Emory University, and a team of investigators conducted the RESET trial, a phase 3, randomized, double-blind, placebo-controlled study of the efficacy and safety of rivipansel for VOC requiring hospitalization. Included were 345 patients—204 patients at least 18 years old and 141 patients 6-17 years old. Patients were randomized to an intravenous rivipansel loading dose, followed by up to 14 additional doses every 12 hours, or placebo, in addition to standard care. A total of 162 patients were treated with rivipansel and 158 patients were treated with placebo.

The primary study endpoint was time to readiness for discharge. Additional endpoints were time to discharge, time to discontinuation of IV opioids, and cumulative IV opioid use.

The study did not show statistically significant improvements in outcomes for the total population, although rivipansel treatment within 26.4 hours of pain onset reduced median time to readiness for discharge by 56.3 hours from 122 to 65.7 hours compared to placebo. It also reduced median time to discharge by 41.5 hours from 112.8 to 71.3 hours and reduced median time to discontinuation of IV opioids by 50.5 hours from 104 to 53.5 hours, compared to placebo.

In the population of patients aged 6-17 years old, the benefit with rivipansel depended on the duration of VOC prior to treatment. Those who were treated with rivipansel within 30 hours of the onset of VOC experienced a reduction in median time to readiness for discharge by 29.3 hours from 94.1 to 64.8 hours, reduction in median time to discharge by 23.2 hours from 92.8 to 69.6 hours, and reduction in median time to discontinuation of IV opioids by 15.4 hours from 68.9 to 53.5 hours. Early use of rivipansel decreased median time to readiness for discharge by more than 1 day and resulted in more children ready for discharge by 24, 48, and 72 hours, compared to those who received placebo.

Pain scale assessments demonstrated a substantial reduction in the time to the first clinically meaningful reduction in pain.

The findings could change the VOC treatment paradigm for patients with VOC in sickle cell disease.

Early Initiation of Treatment with Rivipansel for Acute Vaso-Occlusive Crisis in Sickle Cell Disease (SCD) Achieves Earlier Discontinuation of IV Opioids and Shorter Hospital Stay: Reset Clinical Trial Analysis

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Early initiation reduces hospital stays and length of IV opioid use.

Early Initiation of Rivipansel for VOC in Sickle Cell Disease

A mobile health (mHealth) app may help children manage pain and symptoms related to 

, according to findings presented at the 

Shannon Phillips, PhD, RN, College of Nursing, Medical University of South Carolina, and a team of investigators developed a mHealth self-management intervention for patients with pain related to their sickle cell disease. They included 60 dyads of children with sickle cell disease up to 18 years old and the parent/caregiver.

Data collected came from app use, child and parent/caregiver surveys at baseline, mid-intervention, end of the intervention, and post-intervention. PROMIS SF was used to measure anxiety, fatigue, depressive symptoms, pain interference, pain intensity, and sleep disturbance, while PedsQL and Sickle Cell Disease Module were used to measure health-related quality of life.

The intervention, Voice Crisis Alert V2, was developed with an end-user-based approach and guided by the Pediatric Self-Management model. Components of the digital health app included “Crisis Care,” “I’m in Pain,” “Pain History,” “Messaging,” and “Sickle Cell Information.” Users could document their health history, record pain, and speak securely with a provider through the app. The team tested the feasibility of the digital health tool on symptoms and quality of life.

Children ages 8-17 years old experienced the most substantial pre- to post-intervention differences in symptoms and quality of life scores. The scores represented an improvement for all symptom outcomes except anxiety, with clinically significant improvements in depressive symptoms, fatigue, and pain interference. The team noted improvements in total scores and all subscale scores on the PedsQL and Sickle Cell Disease Module instruments.

Frequency and length of app use were not correlated with the degree of outcome improvements.

On the Crisis Care component of the app, negative correlations were observed between the number of times it was used and the treatment score (

s = -46, 95% CI [-.75 to -.00]), the number of minutes it was used and the treatment score (

s = -.54, 95% CI [-.80 to -.11]), and the number of minutes the Crisis Care component was used and the total Sickle Cell Disease Module score (

s = -.48, 95% CI [-.77 to -.03]). Such correlations suggested more frequent and longer use of the Crisis Care component was associated with less substantial improvement in Sickle Cell Disease Module total score and treatment score.

Based on the results, the app could be best suited for children ages 8-17 years old compared to children less than 8 years old with sickle cell disease. Specifically, the use of the app may lead to an improvement in fatigue, pain interference, health-related quality of life, physical functioning, pain and hurt, pain impact, pain management, and treatments.

Assessment of Impact of a mHealth Self-Management Intervention for Children with Sickle Cell Disease

The app improved pain, fatigue, and quality of life in children with the disease.

mHealth App Improves Sickle Cell Disease Symptoms

 in patients with hematological malignancies, according to study findings presented at the 

62nd American Society of Hematology (ASH) Annual Meeting and Exposition

The findings suggested the need to establish the diagnosis of COVID-19 quickly, as faster initiation of treatment was associated with better outcomes.

Alex Niu, MD, Section of Hematology and Medical Oncology, Department of Medicine, Tulane University, and a team of investigators examined 29 patients with a variety of hematological malignancies with high suspicion for COVID-19 based on clinical presentation, lab results, and imaging. Patients, aged 24-82 years old, were tested with CRISPR and/or RT-PCR based techniques.

Those included had a variety of hematological malignancies—20 lymphoid and 9 myeloid. Of the patients, 24 presented with an undiagnosed respiratory illness and 5 presented while asymptomatic for testing prior to chemotherapy. All patients were evaluated for COVID-19.

Among the patients, 16 tested positive for COVID-19 with guideline-directed nasopharyngeal RT-PCR testing, including 5 asymptomatic. There were 13 patients who tested negative but based on their clinical history, imaging, and disease course, there was still concern the patients were infected.

The team used CRISPR technology to test 8 patients who initially tested negative by RT-PCR. Of those re-tested, 7 of the 8 patients tested positive for COVID-19 with a blood sample and/or nasal swab for the SARS-CoV-2 specific N gene and ORF1ab gene. Without the patients who were negative and not tested by CRISPR, the rate of false negativity with RT-PCR testing was elevated at 29% in the team’s cohort of hematological malignancies.

There was a very high fatality rate observed, with 31% of patients dying. For the 23 patients who tested positive, 8 patients received COVID-19-directed therapy with either hydroxychloroquine/azithromycin, remdesivir, and/or COVID-19 convalescent plasma, and 4 patients died. Of the 8 patients, 7 improved and 1 died.

Among the 5 patients who were negative with no CRISPR completed, 1 received hydroxychloroquine/azithromycin due to symptoms and imaging and recovered. Three patients died outside the facility due to unknown causes.

A majority of the patients included in the study had undergone SCT or were actively on chemotherapy. Most were on lymphodepleting chemotherapy. Symptoms and immune response to COVID-19 were likely to differ from immunocompetent patients, which resulted in an overall worse outcome in the patients. The 5 asymptomatic patients had lymphoid malignancies and were on chemotherapy.

For patients with hematological malignancies, investigators noted the need to establish an algorithm to diagnose COVID-19 efficiently. Treatment pathways must also be instituted to treat the infection and provide the best treatment for the underlying hematological malignancies.

COVID-19 in Patients with Hematological Malignancies: High False Negative Rate with High Mortality

,” was presented at the 62nd ASH Annual Meeting and Exposition.

Faster initiation of COVID-19 treatment is associated with better outcomes in patients with hematological malignancies. 

High COVID-19 False-Negative Rate Among Patients With Hematological Malignancies

A mobile- and web-based health intervention program for 

 and prediabetes management may be a solution for helping patients with the disease.

The program, which was generally well-received by patients, saw high engagement that gradually decreased. Adherence to the program, individualized programs, and technical barriers needed to be addressed to maximize potential health benefits.

Virginia Signal, PhD, and a team of investigators used the BetaMe/Melon digital health intervention for people with type 2 diabetes or prediabetes. The program lasted 12 months and had an initial 16-week active support phase of 4 interventions, health coaching, goal setting and tracking, online peer support in a secure forum, and provision of evidence-based resources. The final 36 weeks used online peer support and goal tracking only.

The team conducted a randomized controlled trial and compared the outcomes of patients aged 18-75 years old with HbA1c of 41-70 mmol/mol enrolled in the program or receiving usual primary health care. The investigators recruited 429 participants, with 215 randomly placed to the intervention arm and 214 allocated to the control arm.

Data were collected from the mobile/web platform, an online questionnaire completed during the final study assessment, and telephone interviews conducted with a subset of participants at the end of the study period. For the questionnaire, participants were asked about the usefulness of the full 12-month program and whether they would recommend it to others. There were 18 phone interviews conducted by a researcher to get in-depth perceptions about the most and least useful components of the program, barriers to incorporating the program into daily life and suggested changes to the program.

Primary study outcomes included mean changes in HbA1c and weight from baseline to 12 months.

Of the intervention participants, 92% received an initial health coaching session, while 74% (95% CI, 68-80.1) were actively engaged at some point during the 16-week active support phase. The team noted women were more likely to actively engage at any time (82.4%) than men (66.4%).

Overall, there was a steady decline in the usage of the digital health program over the 16-week support phase, from 50% of participants engaging at week 0 down to 22.3% at week 15. Participants in the youngest age group (35-54 years old) had higher engagement than other age groups. Still, by around week 6, the engagement rates converged with those of other age groups.

Diary entries were the most common and frequent form of engagement by all participants.

Nearly 84% of intervention arm participants completed the online questionnaire. Participants rated the education resources and health coaches as more useful components of the program, with 63.7% and 59.2% either agreeing or strongly agreeing with such statements. Goal tracking and online peer support were viewed as less useful, while the overall program was seen as easy to use. More than 60% of participants noted they would recommend the program to friends and family.

Participants who were interviewed by phone mostly indicated they had a positive experience and were able to increase their knowledge of diabetes or prediabetes, resulting in behavior change. Conversely, some participants had difficulties connecting or staying connected to their health coach. Similar to the results of the online questionnaire, participants found the online peer support and goal tracking to be the least useful components.

While the program was generally well-received by patients, it will be important to learn how to improve steady engagement and get the most out of such a digital health intervention.

A Mobile- and Web-Based Health Intervention Program for Diabetes and Prediabetes Self-Management (BetaMe/Melon): Process Evaluation Following a Randomized Controlled Trial

A digital health program may be effective for patients with diabetes or prediabetes.