About this Research Topic
Diseases defined as thrombotic microangiopathies (TMA) encompass a variety of distinct pathologic
presentations that in many clinical settings can prove challenging to treat. This challenge can be
greater due to overlapping symptomatology and ambiguous laboratory results that can make
discerning a likely single entity responsible for a patient's presentation elusive. Moreover, in other
instances diagnosis of exclusion may prove the only option available to guide a clinician which is not
ideal. This is important since each of these etiologies responds to different therapies with a distinct
road to recovery.
Over the last two decades, a deluge of information has become available once molecular targets that
lead to pathology were identified, such as ADAMTS13 in the setting of thrombotic thrombocytopenic
purpura (TTP), dysregulation of the complement pathway, and treatments such as the discovery and
application of the anti-C5 monoclonal antibody eculizumab for the treatment of atypical hemolytic
uremic syndrome (aHUS), and just this year caplacizumab approval for TTP treatment. These
discoveries have changed the way not only of how these diseases are treated but have also resulted in
appropriate therapies being used earlier in the clinical course leading to better outcomes. For
example, one of these changes is reserving therapeutic plasma exchange for those patients with
ADAMTS13 deficiency who represent the patient group that mostly benefits from this therapy within
the TMA disease group. Yet other diseases in which pathology leads to significant thrombocytopenia
secondary to either autoimmune processes such as systemic lupus erythematosus and vasculitis,
metastatic malignancies that compromise bone marrow output, or severe hypertension among
others can confuse the clinical picture due to similarities with TTP causing delays in recognition and
initiation of appropriate therapy that will lead to the restoration of platelet counts that does not involve
TPE.
The aim of this Research Topic is to be a forum to present novel research from basic science to
translational and clinical work, and when applicable reviews of available data that puts each of these
diseases in the context of the latest discoveries. In regard to original research, works that aim to
describe new disease targets, description of potential pathologic pathways, promising therapeutic
approaches, and validation of diagnostic algorithms are welcome. Likewise, research that helps to
differentiate earlier etiologies unlikely to benefit from TPE is also welcome.
Details for authors: Investigators working on animal models of TTP, aHUS, HUS among others or
clinical studies in these areas are encouraged to submit their work. Furthermore, authors looking at
novel laboratory tests or new ways of using currently available ones that raise suspicion of a given
etiology over others are similarly welcome. Likewise, research shedding light at mechanisms leading
to microangiopathic hemolytic anemias secondary to a primary insult are equally welcome. We do
not encourage the submission of case reports unless they are in the context of a comprehensive review
of relevant literature that may lead to changes in practice and treatment approaches.
presentations that in many clinical settings can prove challenging to treat. This challenge can be
greater due to overlapping symptomatology and ambiguous laboratory results that can make
discerning a likely single entity responsible for a patient's presentation elusive. Moreover, in other
instances diagnosis of exclusion may prove the only option available to guide a clinician which is not
ideal. This is important since each of these etiologies responds to different therapies with a distinct
road to recovery.
Over the last two decades, a deluge of information has become available once molecular targets that
lead to pathology were identified, such as ADAMTS13 in the setting of thrombotic thrombocytopenic
purpura (TTP), dysregulation of the complement pathway, and treatments such as the discovery and
application of the anti-C5 monoclonal antibody eculizumab for the treatment of atypical hemolytic
uremic syndrome (aHUS), and just this year caplacizumab approval for TTP treatment. These
discoveries have changed the way not only of how these diseases are treated but have also resulted in
appropriate therapies being used earlier in the clinical course leading to better outcomes. For
example, one of these changes is reserving therapeutic plasma exchange for those patients with
ADAMTS13 deficiency who represent the patient group that mostly benefits from this therapy within
the TMA disease group. Yet other diseases in which pathology leads to significant thrombocytopenia
secondary to either autoimmune processes such as systemic lupus erythematosus and vasculitis,
metastatic malignancies that compromise bone marrow output, or severe hypertension among
others can confuse the clinical picture due to similarities with TTP causing delays in recognition and
initiation of appropriate therapy that will lead to the restoration of platelet counts that does not involve
TPE.
The aim of this Research Topic is to be a forum to present novel research from basic science to
translational and clinical work, and when applicable reviews of available data that puts each of these
diseases in the context of the latest discoveries. In regard to original research, works that aim to
describe new disease targets, description of potential pathologic pathways, promising therapeutic
approaches, and validation of diagnostic algorithms are welcome. Likewise, research that helps to
differentiate earlier etiologies unlikely to benefit from TPE is also welcome.
Details for authors: Investigators working on animal models of TTP, aHUS, HUS among others or
clinical studies in these areas are encouraged to submit their work. Furthermore, authors looking at
novel laboratory tests or new ways of using currently available ones that raise suspicion of a given
etiology over others are similarly welcome. Likewise, research shedding light at mechanisms leading
to microangiopathic hemolytic anemias secondary to a primary insult are equally welcome. We do
not encourage the submission of case reports unless they are in the context of a comprehensive review
of relevant literature that may lead to changes in practice and treatment approaches.
Keywords: thrombotic microangiopathies, TMA, ADAMTS13, thrombotic thrombocytopenic purpura, atypical hemolytic uremic syndrome, TTP, aHUS, HUS
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