A 56-year-old woman presented with dermatomyositis positive for anti-melanoma differentiation-associated gene 5 antibody. No interstitial lung disease was detected. Despite treatment with methylprednisolone pulse therapy and cyclosporine, dysphagia developed. Furthermore, the presence of thrombocytopenia, elevated lactate dehydrogenase levels, and an undetectable haptoglobin level suggested the possibility of thrombotic microangiopathy (TMA). Disturbed consciousness developed shortly after TMA onset, and brain magnetic resonance imaging revealed hyperintensity lesions in the bilateral basal ganglia, thalami, and brainstem. The patient was diagnosed with atypical posterior leukoencephalopathy syndrome before dying of heart failure later that day. In conclusion, early TMA recognition and prompt intensive treatment are critical in such cases.
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http://dx.doi.org/10.2169/internalmedicine.7309-21 | DOI Listing |
Hum Gene Ther
January 2025
BridgeBio Gene Therapy, Palo Alto, California, USA.
Complement-mediated thrombotic microangiopathy (TMA) in the form of atypical hemolytic uremic syndrome (aHUS) has emerged as an immune complication of systemic adeno-associated virus (AAV) gene transfer that was unforeseen based on nonclinical studies. Understanding this phenomenon in the clinical setting has been limited by incomplete data and a lack of uniform diagnostic and reporting criteria. While apparently rare based on available information, AAV-associated TMA/aHUS can pose a substantial risk to patients including one published fatality.
View Article and Find Full Text PDFRev Med Liege
January 2025
Service d'Hématologie clinique, CHU 20 Août, Casablanca, Maroc.
We conducted a retrospective study of 83 cases of immune thrombocytopenia (IT) in patients under 20 years of age. The aim was to provide an overview of IT in our young patients. The median age was 10 years, with a predominance of females (71 %).
View Article and Find Full Text PDFBone Marrow Transplant
January 2025
Division of Pediatric Hematology/Oncology, Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Republic of Korea.
Transplant-associated thrombotic microangiopathy (TA-TMA) is an increasingly recognized complication in hematopoietic cell transplantation (HCT). Given the rarity of prospective pediatric studies on TA-TMA, this study aimed to evaluate the incidence, survival outcomes, and risk factors for predicting early the development of TA-TMA in a pediatric population following allogeneic HCT. We conducted a prospective analysis of 173 pediatric patients to evaluate the incidence, survival outcome, and risk factors of TA-TMA.
View Article and Find Full Text PDFTransplant Cell Ther
January 2025
Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH; Department of Pediatrics, University of Cincinnati, Cincinnati, OH.
Background: HSCT conditioning regimens cause massive lysis of hematopoietic cells with release of toxic intracellular molecules into the circulation.
Objectives: To describe the response to oxidative stress early after hemopoietic stem cell transplantation (HSCT) and assess the association of early oxidative stress with later transplant outcomes.
Study Design: Key components of in the body's physiological response to oxidative stress were studied in a cohort of 122 consecutive pediatric allogeneic HSCT recipients.
J Immunother Precis Oncol
February 2025
Section of Benign Hematology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Thrombotic thrombocytopenic purpura (TTP) is characterized by thrombotic microangiopathy resulting from decreased activation of the von Willebrand factor-cleaving protease (ADAMTS13). TTP can cause organ damage and is often fatal if the appropriate treatment is not started immediately. Although primary immune TTP is the most common form of TTP, secondary immune etiologies, including complications from immune checkpoint inhibitors (ICIs), have also been reported.
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