Clinical trial NCT05649280: Safety Evaluation of DREPADOM - Home Care Services and Hospitalizations for Sickle Cell Disease Patients
Official title: Safety Evaluation of DREPADOM - Home Care Services and Hospitalizations for Sickle Cell Disease Patients
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Sickle Cell Disease
Lead sponsor: Soutien aux Actions contre les Maladies du Globule Rouge
Sponsor class: OTHER
First posted: 2022-12-14
Enrollment: 100
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Sickle Cell Disease (SCD) is the most widespread genetic disease in the world. With an incidence rate of approximately 1/3000 newborns each year in France, International Standards consider SCD a rare disease. However, in the Ile-de-France region, that incidence drops to 1/435 newborns, rendering SCD a major territorial issue. It is estimated that more than 10 000 SCD patients live in this region, which represents approximately 30% of the French SCD population and 10% of the European SCD population.

Vaso-occlusive crisis (VOC) or acute bone pain vaso-occlusion is a characteristic manifestation of SCD and represents the first cause of patients 'hospitalizations. Moreover, 16% of VOCs can develop into a potentially severe complication: the acute chest syndrome (ACS).

Brief summary: The objective of this study is to evaluate the incidence of ACS within the DREPADOM system and compare it to expected incidences of ACS (historic cohorts of PRESEV1 and PRESEV2)

Detailed description: This prospective, observational study will take place, as follows:

Calculation of the PRESEV score in the ER will determine or not eligibility of the patient in the DREPADOM structure. With a low score, a patient is eligible for home monitoring and after duly provided information, can sign a patient consent for the use of personal medical data.

The coordinating nurse from DREPADOM can then set up a whole network, with the delivery of oxygen supply at the patient's house, dispatch of a medical prescription to a neighboring pharmacy and daily visits from homecare service providers. Both opioids and parenteral hydration and treatments can be prescribed and daily visits, up to three, are programmed. As such, the patient receives a minimum of 1 visit per day from a nurse for monitoring and is provided with a medical oxygen concentrator for home use (3L/min continuously or for at least 15h/day) and parenteral hydration. In addition, patients are called daily by general practitioners or SCD experts, for teleconsultations.

The DREPADOM coordinating nurse is in constant interaction with nurses/providers of homecare services/medical doctors and status and evolution of the patient's global condition is reported both through the constant contact with the coordinating nurse from DREPADOM and in a dedicated software (Link4Life), with monitoring of blood pressure, temperature, respiratory rate, pain, etc... Link4Life is a software tool developed especially for home monitoring. It is adaptable to specific disorders, fully complies with the French Law for Health Data management and allows data extraction. Link4Life has been adapted to SCD in full collaboration with SCD medical experts of the Henri Mondor hospital. It is to be noted that all medical doctors (general practitioners or SCD experts), as well as coordinating and visiting nurses have all access to this application. As such, Link4Life will gather and capture all patient data.

When reporting patients' clinical parameters daily in the dedicated app (controlled by the DREPADOM team of experts), alerts are generated in the software when these are above or below determined thresholds. The medical team of experts from DREPADOM then takes over for daily teleconsultations and medical decisions for pursuing home monitoring or hospital admittance.

Of important note, a patient can be included twice in the DREPADOM study, for two distinct VOCs. Indeed, the PRESEV1 study highlighted that each crisis is unique and should be treated accordingly.

Data capture will be done through the Link4Life software, as well as extraction for data analysis.

* Open-label
* Blind
* Number of treatment arms:

Single arm

Multi arms specify the number of arms:

* Not Controlled
* Controlled, specify the comparator:

Versus placebo Versus reference product Versus other (specify) -Randomized: Parallel group Cross over Other (specify)

-Intervention (gas and/or drug, and/or device, and/or service /study arm):

Eligibility criteria: Inclusion Criteria:

* SS or Sβ0
* Adult patients with a low PRESEV score

Exclusion Criteria:

* SC/Sβ+ patients
* SCD adult patients with an intermediate or high PRESEV score
* Pregnant or breastfeeding women
* Homeless patient
* Patients without Social Security
* Patient deprived of liberty by judicial or administrative decision or patient under guardianship

Primary outcomes:
measure: Incidence of acute chest pain
timeFrame: 30 months
Secondary outcomes:
measure: Incidence of clinical complications associated to Sickle cell disease
timeFrame: 30 months
Other outcomes:
Locations:
city: Creteil
country: France
facility: Henri Mondor Hospital
Study officials:
affiliation: Soutien aux Actions contre les Maladies du Globule Rouge
name: Pablo BARTOLUCCI, PROFESSOR
role: PRINCIPAL_INVESTIGATOR
References: