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Rationale &amp; objective <p>Thrombotic Microangiopathies (TMAs) include an etiological diverse group of phenotypically similar disorders. While individually rare, they are seen as an aggregate with regularity. Prior reports suggested the importance of approaching TMAs in a multidisciplinary fashion. We present the development and data over a 4-year period after establishing the University of California Irvine (UCI) TMA team.</p> Design, settings, participants, and measurements <p>This is a single-center retrospective case observational study of 101 diverse patients demonstrating demographics, diagnoses, triggers, treatments applied, and outcomes representing a wide range of hematologic pathologies.</p> Findings <p>Of the 101 patients, 47 were females with an age range between 18 and 83 years old. 46 of our 101 patients were diagnosed with TMA. 24 patients were diagnosed with atypical hemolytic uremic syndrome (aHUS). Eight patients were diagnosed with Hematopoietic Stem Cell Transplant TMA (HSCT-TMA). Of the 24 aHUS patients, 16 were treated and 8 were evaluated for complement blockade. Of those treated, 100% demonstrated a hematologic response, 81.3% had initial renal remission and 68.8% of those who were on dialysis (11 of 16) remain off dialysis. Of the 8 HSCT-TMA patients, 5 were treated and 3 were evaluated for complement blockade. Of those treated, 80% demonstrated a hematologic response, 100% had initial renal remission; however, of all treated patients eventually returned to needing dialysis.</p> Limitations and conclusions <p>This observational study highlights the importance of the TMA multidisciplinary team to improve diagnosis and optimize outcomes of this rare condition. Future research is needed to subdivide response to various TMA to current and emerging therapies in the context of multidisciplinary care.</p> Clinical trial number <p>Not applicable.</p>

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Thrombotic microangiopathy multidisciplinary assessment team: demographics, final diagnosis, treatment, and outcomes

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年9月26日

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  • Matthew Nguyen,
  • Gayathri Dileep,
  • Samir Patel,
  • Fawaz Al Ammary,
  • Minh-Ha Tran,
  • Benjamin J. Lee,
  • Stefan Ciurea,
  • Sheetal Desai,
  • Alpesh Amin,
  • Hongyu Zhao,
  • Omid Vadpey,
  • Dao Le,
  • Tai Truong,
  • Antoinette Abdelmalek,
  • Jordan Perkins,
  • Rebecca Ahdoot,
  • Fatima Malik,
  • Antoney Ferrey,
  • Uttam Reddy,
  • Ekamol Tantisattamo,
  • Yongen Chang,
  • Wei Ling Lau,
  • Yulian Khagi,
  • Lisa Lee,
  • Zahra Pakbaz,
  • Ramy M. Hanna

摘要

Rationale & objective

Thrombotic Microangiopathies (TMAs) include an etiological diverse group of phenotypically similar disorders. While individually rare, they are seen as an aggregate with regularity. Prior reports suggested the importance of approaching TMAs in a multidisciplinary fashion. We present the development and data over a 4-year period after establishing the University of California Irvine (UCI) TMA team.

Design, settings, participants, and measurements

This is a single-center retrospective case observational study of 101 diverse patients demonstrating demographics, diagnoses, triggers, treatments applied, and outcomes representing a wide range of hematologic pathologies.

Findings

Of the 101 patients, 47 were females with an age range between 18 and 83 years old. 46 of our 101 patients were diagnosed with TMA. 24 patients were diagnosed with atypical hemolytic uremic syndrome (aHUS). Eight patients were diagnosed with Hematopoietic Stem Cell Transplant TMA (HSCT-TMA). Of the 24 aHUS patients, 16 were treated and 8 were evaluated for complement blockade. Of those treated, 100% demonstrated a hematologic response, 81.3% had initial renal remission and 68.8% of those who were on dialysis (11 of 16) remain off dialysis. Of the 8 HSCT-TMA patients, 5 were treated and 3 were evaluated for complement blockade. Of those treated, 80% demonstrated a hematologic response, 100% had initial renal remission; however, of all treated patients eventually returned to needing dialysis.

Limitations and conclusions

This observational study highlights the importance of the TMA multidisciplinary team to improve diagnosis and optimize outcomes of this rare condition. Future research is needed to subdivide response to various TMA to current and emerging therapies in the context of multidisciplinary care.

Clinical trial number

Not applicable.