CAPS or Catastrophic Antiphospholipid Syndrome is an autoimmune and kidney disease which shares the same experience of thrombotic microangiopathy with aHUS/ cTMA.
It is a rare life threatening diseases which can be described as resembling aHUS/cTMA. Also a secondary TMA, though in new nomenclature it would probably be regarded as one of the autoimmune antibody mediated TMAs ( AA-TMA?) or one of the vascular TMAs like Lupus. SLE-TMA.
The TMA can be triggered by the condition itself, as does, Lupus but in some cases complement involvement can follow that and be secondary to the initial CAPS-TMA event. So some CAPS patients may get an aHUS/cTMA diagnosis or even a suspected diagnosis..
BUT it may be more likely that aHUS/cTMA patients may get a CAPS diagnosis or suspected diagnosis first.
The common form APS-Antiphospholipid Syndrome has a prevalence of 400/500 per million so not quite so rare but the catastrophic version is much rarer, only 1% of all APS patients about 4/5 per million. So it is as ultra rare as aHUS/cTMA and presenting similar diagnostic challenges.
Imagine therefore how extremely ultra rare are those who overlap between the two. The level of rareness that makes research into the condition very difficult indeed.
The CAPS-CARE Steering Group are setting out to do just that.
Led by Dr Lucas Jacobs (Brussels University Hospital, Belgium), CAPS-CARE, supported by an international multidisciplinary steering committee and bringing together experts in nephrology, lupus, antiphospholipid syndrome, internal medicine, and intensive care medicine are planning an international survey.
The survey aims to better understand current practices and unmet needs in CAPS diagnosis and management worldwide. This will be the first international survey specifically exploring real-world clinical decision-making in CAPS across specialties and countries.
The study was reviewed and approved by the Ethics Committee of Brussels University Hospital (Hôpital Universitaire de Bruxelles, H.U.B., Belgium).\
Given the significant clinical overlap between CAPS and complement-mediated thrombotic microangiopathies such as aHUS /cTMA, Global Action agreed with Dr Jacobs that this initiative may be of interest to our community.
As CAPS remains an exceptionally rare condition, worldwide participation is essential to achieve a sufficient and truly global representation of current clinical practice. This is the an opportunity for those with a CAPS /aHUS connection to help close knowledge gaps.
Global Action is asking those aHUS/cTMA patients who have had a CAPS diagnosis even if only suspected to let their treating physician know about this study.
A link to the survey can be found HERE.
For others in the aHUS/cTMA patient community it is an example of what more is likely to happen with other conditions in the spectrum of TMAs to establish the science behind more accurate and speedier diagnosis. Remembering that aHUS/cTMA is at the bottom of a diagnosis by exclusion process.
More information for those who may wish to participate and help the another TMA initiative follows:
CAPS-CARE STUDY SYNOPSIS
TITLE
Catastrophic Antiphospholipid Syndrome – Challenges in Assessment, Recognition and
Evaluation (CAPS-CARE)
International anonymous survey of clinicians regarding the diagnosis and management of
catastrophic antiphospholipid syndrome.
Principal Investigator
Dr Lucas Jacobs
Department of Nephrology and Dialysis
Brussels University Hospital (H.U.B.) – Erasme Hospital
Brussels, Belgium
STEERING COMITEE
Lucas Jacobs, M.D. 1,4*
- Johann Morelle, M.D., Ph.D 2
- Fabio Taccone, M.D., PhD 3
- Filippo Annoni, M.D., Ph.D 3
- Frédéric Vandergheynst, M.D., PhD 4
- Frédéric Houssiau, M.D., Ph.D 5
- Farah Tamirou, M.D., Ph.D 5
- Nader Wauters 6
- Maxime Taghavi, M.D.1
- Brugmann University Hospital, Université libre de Bruxelles (ULB), Brussels, Belgium.
- CHR Namur, Université catholique de Louvain (UCLouvain), Louvain-la-Neuve, Belgium.
- Hôpital Universitaire de Bruxelles (HUB) site Erasme, ULB, Brussels, Belgium.
- HUB site Erasme, ULB, Brussels, Belgium.
- Cliniques Universitaires de Saint-Luc, UCLouvain, Louvain-La-Neuve, Belgium.
- Tivoli University Hospital, ULB, La Louvière, Belgium.
- Lead investigator
2
Coordinating Centre
Brussels University Hospital (H.U.B.), Brussels, Belgium
FUNDING
No external funding.
BACKGROUND AND RATIONALE
Catastrophic antiphospholipid syndrome (CAPS) is a rare but life-threatening manifestation of
antiphospholipid syndrome characterized by rapidly progressive multiorgan thrombosis and
high mortality despite advances in treatment. Approximately 500 cases have been reported in
the international CAPS Registry.
Although international recommendations emphasize the importance of early recognition and
prompt initiation of combined therapy (anticoagulation, corticosteroids, plasma exchange
and/or intravenous immunoglobulins), little is known about how these recommendations are
applied in real-world clinical practice. Diagnostic uncertainty, overlap with thrombotic
microangiopathies, disseminated intravascular coagulation, sepsis, and other critical illnesses
frequently complicate decision-making.
CAPS-CARE was designed to evaluate current clinical practices, diagnostic confidence, and
barriers to optimal management among clinicians likely to encounter CAPS in routine practice.
OBJECTIVES
Primary Objective
To describe current diagnostic and therapeutic practices among clinicians facing confirmed or
suspected catastrophic antiphospholipid syndrome (CAPS).
Secondary Objectives
Assess clinicians’ diagnostic and therapeutic confidence regarding CAPS.
Identify factors associated with hesitation to initiate full treatment in probable CAPS.
Compare practices across specialties and levels of experience.
Identify perceived barriers to diagnosis and treatment.
Estimate delays between CAPS suspicion and initiation of optimal therapy.
STUDY DESIGN
3
International, multicentre, anonymous, non-interventional survey.
The study is conducted through an online questionnaire distributed internationally via scientific
societies, professional networks, academic mailing lists, and expert groups. Responses are
collected anonymously using the REDCap platform.
No patients are included in the study and no clinical intervention is performed.
TARGET POPULATION
Physicians practicing in:
Nephrology
Rheumatology
Internal Medicine
Intensive Care Medicine
Hematology
Participants must be specialists who may encounter suspected or confirmed CAPS during
their clinical practice.
ENDPOINTS
Primary Endpoint
Description of diagnostic and therapeutic approaches adopted by clinicians when facing definite
or probable CAPS.
Secondary Endpoints
Diagnostic and therapeutic confidence levels.
Factors associated with reluctance to treat probable CAPS.
Differences in practice according to specialty, geographical region, and experience.
Identification of diagnostic and organizational barriers.
Estimated time from suspicion of CAPS to treatment initiation.
ETHICS
The study has received approval from the Ethics Committee of Brussels University Hospital
(Université libre de Bruxelles, Belgium).
The survey is entirely anonymous. No patient data and no identifiable participant data are
collected. Participation is voluntary. The study complies with applicable ethical and data
protection regulations, including GDPR requirements.
4
Given its anonymous and non-interventional nature, the study poses no medical or physical risk
to participants.
DISSEMINATION AND AUTHORSHIP
Results will be presented at international scientific meetings and submitted for publication in
peer-reviewed journals.
Clinicians who agree to be identified will be invited to join the CAPS-CARE Study Group and
may contribute to future analyses, scientific communications, and publications arising from the
project.
Collaborating scientific societies supporting dissemination of the survey will be acknowledged
in resulting communications and publications.
WEB LINKS
Survey link: https://redcap.ctc1.hubruxelles.be/surveys/?s=MDXPPDXDCM88DEHF
Lucas Jacobs, M.D.
Article No 804
