Shared Decision-Making Toolkit for Sickle Cell Disease

(EMPOWER-AYA Trial)

Overseen By Clinical Research Coordinator

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Nemours Children's Clinic

Disqualifiers: Seizures, Cognitive impairment, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

68 Participants Needed

Trial Summary

Do I need to stop my current medications for this trial?

The trial information does not specify whether you need to stop taking your current medications. It seems to focus on decision-making tools rather than medication changes.

What data supports the effectiveness of the Shared Decision Making Intervention treatment for sickle cell disease?

Research shows that shared decision-making (SDM) can improve health outcomes for children with sickle cell disease by involving them and their parents in treatment decisions. A study found that using a decision aid for hydroxyurea, a drug for sickle cell anemia, improved knowledge and reduced decisional conflict, suggesting that SDM can help in making informed treatment choices. ^{1 2 3 4 5}

Is the Shared Decision-Making Toolkit for Sickle Cell Disease safe for humans?

The research articles do not provide specific safety data for the Shared Decision-Making Toolkit itself, but they focus on improving decision-making processes for treatments like hydroxyurea in sickle cell disease. There is no mention of safety concerns related to the decision-making process itself. ^{1 3 6 7 8}

How does the Shared Decision Making Intervention treatment differ from other treatments for sickle cell disease?

The Shared Decision Making Intervention is unique because it focuses on involving patients and their families in the decision-making process about their treatment options, rather than just prescribing a specific medical treatment. This approach aims to improve health outcomes by ensuring that treatment decisions align with the patient's values and preferences. ^{1 2 3 9 10}

What is the purpose of this trial?

This study will evaluate the acceptability, feasibility, and preliminary efficacy of a shared decision making intervention for adolescents and young adults (AYAs) with sickle cell disease (SCD). 60 AYAs with SCD ages 15-25 and their caregivers and 8 SCD providers will participate in the pilot pragmatic trial. AYAs, caregivers, and providers will be recruited from Nemours Children's Hospital, Delaware (NCH-DE), Nemours Children's Hospital in Orlando, FL (NCH-ORL), and Nemours Children's Health at Wolfson Children's Hospital in Jacksonville, FL (NCH-JAX). NCH-DE participants (n=30) will receive the SDM intervention including a virtual reality patient health education component, whereas NCH-ORL and NCH-JAX participants (n=30) will receive the SDM intervention with standard patient education materials (print, video). SCD providers will be trained to use the toolkit components and will introduce decision aids during an outpatient clinic visit for AYAs who are candidates for one or more disease-modifying therapies.

Eligibility Criteria

This trial is for adolescents and young adults aged 15-25 with sickle cell disease (SCD), along with their caregivers, who are fluent in English. Participants must be receiving treatment at Nemours or ChristianaCare and considered by their healthcare provider as candidates for new SCD treatments.

Inclusion Criteria

Nemours SCD healthcare providers will also be included.

English language fluency is a requirement for all participants.

I am 15-25 years old with SCD and eligible for new treatments.

Exclusion Criteria

Patients/caregivers who previously participated in usability testing of the intervention will be excluded.

Providers still completing training will be excluded.

I do not have a history of seizures, can complete interviews, and if under 18, have a guardian for consent.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Intervention

Participants receive the shared decision making intervention, including either virtual reality or standard patient education materials

1 visit

1 visit (in-person)

Follow-up

Participants are monitored for acute healthcare utilization and satisfaction with the intervention

6 months

Treatment Details

Interventions

• Shared Decision Making Intervention

Trial Overview The study tests a shared decision-making toolkit designed to help patients with SCD make informed choices about their treatment. Some will use the toolkit with virtual reality education, while others will have standard materials like print and video.

Participant Groups

Group I: Shared Decision Making Toolkit without VR Experimental Treatment 1 Intervention

Group II: Shared Decision Making Toolkit including VR Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Nemours Children's Clinic

Lead Sponsor

Trials

128

Recruited

18,000+

Findings from Research

In a study of 24 consultations involving children with sickle cell disease, the objective measurement of shared decision-making (SDM) was low, with a median score of only 25%, indicating significant room for improvement in involving patients and their families in treatment decisions.

While subjective perceptions of SDM were fair, there is a notable gap between paediatricians' confidence in their ability to engage families and the actual experience of children and parents, highlighting the need for better training and interventions to enhance SDM practices.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Shared decision-making between paediatric haematologists, children with sickle cell disease and their parents: an exploratory study. Wijngaarde, R., Koning, M., Fijnvandraat, K., et al.[2023]

A qualitative study involving 36 physician interviews revealed two main approaches to decision-making in sickle cell disease treatments: the Collaborative approach, which emphasizes discussing all treatment options with patients, and the Proponent approach, which advocates for a specific treatment plan to persuade patients.

The study highlights the complexity of treatment decisions for sickle cell disease, influenced by patient, disease, and physician factors, suggesting a need for systems that enhance patient engagement and shared decision-making in treatment planning.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Proponent or collaborative: Physician perspectives and approaches to disease modifying therapies in sickle cell disease. Bakshi, N., Sinha, CB., Ross, D., et al.[2022]

A multicomponent decision aid for hydroxyurea was developed to help parents and clinicians discuss treatment options for children with sickle cell anemia, involving input from 75 parents and 28 clinicians.

The use of this decision aid improved parents' knowledge about hydroxyurea and reduced their decisional conflict, indicating it could effectively support shared decision-making in pediatric care.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Development of a Hydroxyurea Decision Aid for Parents of Children With Sickle Cell Anemia. Crosby, LE., Walton, A., Shook, LM., et al.[2020]

Patients with sickle cell disease (SCD) consider multiple factors, including lifestyle and health status, when deciding whether to take hydroxyurea (HU), highlighting the complexity of treatment decisions.

Effective communication from healthcare providers, particularly through shared decision-making, significantly influences patients' willingness to engage with HU treatment; those who felt unheard were more likely to disengage from their treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The Role of Patient-Physician Communication on the Use of Hydroxyurea in Adult Patients with Sickle Cell Disease. Jabour, SM., Beachy, S., Coburn, S., et al.[2020]

A qualitative study involving 20 interviews with caregivers and individuals with sickle cell disease (SCD) revealed that decision-making around opioid use for pain management is complex and requires collaboration among patients, families, and healthcare providers.

The study identified key themes that can enhance shared decision-making strategies in clinical settings, emphasizing the importance of considering personal values, psychological states, and the context of care when discussing opioid therapy for pain management in SCD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Patient and Family Opioid Decision-Making for Pain Management in Sickle Cell Disease: A Qualitative Study. Phillips, S., Schlenz, AM., D'Alton, S., et al.[2023]

Patients with sickle cell disease and their caregivers feel that while healthcare provider education is helpful, it does not fully meet their learning needs, leading them to seek additional information independently.

The study identified that improving quality of life is a primary concern for patients and caregivers when considering treatment options, and they expressed a preference for peer experiences as a source of information, highlighting the potential benefit of a web-based decision aid to support their educational needs.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Educational needs of patients and caregivers living with sickle cell disease results in development of web-based patient decision aid. Ross, D., Sinha, C., Bakshi, N., et al.[2021]

A survey of 63 primary care providers in North Carolina revealed that 64% care for 1 to 10 patients with sickle cell disease, but only 39% regularly communicate with SCD specialists, indicating a gap in co-management practices.

Providers showed low awareness and usage of the SCD Toolbox, which is designed to aid in SCD management, and expressed a preference for accessing the toolbox in various formats such as a website, mobile app, or paper.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Awareness and Use of the Sickle Cell Disease Toolbox by Primary Care Providers in North Carolina. Ibemere, SO., Tanabe, P., Bonnabeau, E., et al.[2021]

A series of workshops and educational sessions were organized to empower sickle cell disease (SCD) patients in Europe, allowing them to influence research priorities and enhance their involvement in the scientific community.

These initiatives have improved patient experience and empowerment, benefiting not only patients and caregivers but also health professionals, highlighting the importance of patient-centered approaches in medical research.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Sickle cell disease: embedding patient participation into an international conference can transform the role of lived experience. Pellegrini, M., Chakravorty, S., Del Mar Manu Pereira, M., et al.[2023]

Shared decision-making (SDM) is crucial in pediatric care, as it involves collaboration between patients, families, and healthcare providers to make informed health decisions, yet it remains underutilized despite evidence of its benefits.

Insights from a pediatric patient, a parent, and a pediatrician highlight the positive impacts of SDM on care outcomes and emphasize the need for practical recommendations to enhance its adoption in clinical practice.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Is sharing really caring? Viewpoints on shared decision-making in paediatrics. Jordan, Z., Tremblay, C., Lipstein, E., et al.[2021]

The study assessed the level of shared decision making (SDM) in discussions about hematopoietic stem cell transplantation (HSCT) among 26 patients with sickle cell disease (SCD) and thalassemia, finding a moderate SDM score of 43%.

While patients were informed about treatment options, the process primarily involved one-way communication, indicating a need for improvement in actively engaging patients in discussions about their preferences and treatment choices.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Decision making for hematopoietic stem cell transplantation in pediatric, adolescent, and young adult patients with a hemoglobinopathy-Shared or not? Mekelenkamp, H., Smiers, F., Camp, N., et al.[2022]

References

1. Germany pubmed.ncbi.nlm.nih.gov

Shared decision-making between paediatric haematologists, children with sickle cell disease and their parents: an exploratory study. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Proponent or collaborative: Physician perspectives and approaches to disease modifying therapies in sickle cell disease. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Development of a Hydroxyurea Decision Aid for Parents of Children With Sickle Cell Anemia. [2020]

4. Switzerland pubmed.ncbi.nlm.nih.gov

The Role of Patient-Physician Communication on the Use of Hydroxyurea in Adult Patients with Sickle Cell Disease. [2020]

5. United States pubmed.ncbi.nlm.nih.gov

Patient and Family Opioid Decision-Making for Pain Management in Sickle Cell Disease: A Qualitative Study. [2023]

6. England pubmed.ncbi.nlm.nih.gov

Educational needs of patients and caregivers living with sickle cell disease results in development of web-based patient decision aid. [2021]

7. United States pubmed.ncbi.nlm.nih.gov

Awareness and Use of the Sickle Cell Disease Toolbox by Primary Care Providers in North Carolina. [2021]

8. England pubmed.ncbi.nlm.nih.gov

Sickle cell disease: embedding patient participation into an international conference can transform the role of lived experience. [2023]

9. Australia pubmed.ncbi.nlm.nih.gov

Is sharing really caring? Viewpoints on shared decision-making in paediatrics. [2021]

10. United States pubmed.ncbi.nlm.nih.gov

Decision making for hematopoietic stem cell transplantation in pediatric, adolescent, and young adult patients with a hemoglobinopathy-Shared or not? [2022]