Recovery from life-threatening transplantation-associated thrombotic microangiopathy using eculizumab in a patient with very severe aplastic anemia

Okano, Munehiro; Sakata, Naoki; Ueda, Shinichiro; Takemura, Tsukasa

doi:10.1038/bmt.2014.97

Cited by 17 publications

(30 citation statements)

References 8 publications

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“…A faster rate of improvement was seen when medication was given promptly after diagnosis, which may also prevent irreversible end organ damage. A higher dose of eculizumab is required to treat TMA associated with HSCT when compared to giving it for aHUS, aiming for therapeutic blood levels of >99 μg/mL . Despite the high dose required it has a low toxicity …”

Section: Thrombotic Microangiography Following Chemotherapymentioning

confidence: 99%

Hematopoietic stem cell transplantation and acute kidney injury in children: A comprehensive review

Raina

Herrera

Krishnappa

et al. 2017

Pediatric Transplantation

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AKI in the setting of HSCT is commonly investigated among adult patients. In the same way, malignancies requiring treatment with HSCT are not limited to the adult patient population, AKI following HSCT is frequently encountered within pediatric patient populations. However, inadequate information regarding epidemiology and pathophysiology specific to pediatric patients prevents development of appropriate and successful therapeutic strategies for those afflicted. Addressing AKI in the context of sinusoidal obstruction syndrome, chemotherapy, thrombotic microangiopathy and hypertension post chemotherapy, glomerulonephritis, and graft versus host disease provides greater insight into renal impairment associated with these HSCT-related ailments. To obtain a better understanding of AKI among pediatric patients receiving HSCT, we investigated the current literature specifically addressing these areas of concern.

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Section: Thrombotic Microangiography Following Chemotherapymentioning

confidence: 99%

Hematopoietic stem cell transplantation and acute kidney injury in children: A comprehensive review

Raina

Herrera

Krishnappa

et al. 2017

Pediatric Transplantation

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“…In a report by Okano et al, a total of 14 doses of eculizumab were used till TA-TMA symptoms resolved. 72 Jodele et al reported a median use of 5.5 doses (range 2-21) in patients who responded to treatment. 13 Eculizumab therapy is associated with an increased susceptibility to encapsulated organisms, so Figure 3.…”

Section: Managementmentioning

confidence: 99%

“…13 A review of published reports 65 demonstrates the use of eculizumab in a total of 26 cases of TA-TMA following HSCT or solid organ transplant. 9,13,[66][67][68][69][70][71][72][73][74][75] The cases were initially treated with discontinuation or dose reduction in CNI with or without plasma exchange. Of these patients, 24 (92%) were alive at 1-year follow-up, 65 and the remaining 2 (8%) pediatric patients did not achieve therapeutic levels even after dose escalation and died.…”

Section: Managementmentioning

confidence: 99%

Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy

Elsallabi

Bhatt

Dhakal

et al. 2015

Clin Appl Thromb Hemost

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Hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA) is a fatal, multifactorial disorder, which may present with thrombocytopenia, hemolysis, acute renal failure, mental status changes and involvement of other organs. The pathogenesis of TA-TMA is complex and includes multiple risk factors such as certain conditioning regimens, calcineurin inhibitors (CNIs), graft-versus-host disease (GVHD), human leukocyte antigen mismatch, and opportunistic infections. The end result of these insults is endothelial injury in the kidney and other organs. Recent studies also indicate a role of complement activation in tissue damage. The lack of sensitive and specific diagnostic tests for TA-TMA often results in delayed diagnosis. Biopsy is not always possible for diagnosis because of the risk of complications such as bleeding. Recently, an emerging role of renal-centered screening approach has been demonstrated, which utilize the monitoring of blood pressure, urine protein, serum lactate dehydrogenase and hemogram for early detection. Therapeutic options are limited, and plasma exchange plays a minor role. Withdrawal of offending agent such as CNIs and the use of rituximab can be effective in some patients. However, the current treatment strategy is suboptimal and associated with high mortality rate. Recently, eculizumab has been utilized in a few patients with good outcomes. Patients, who develop TA-TMA, are also at an increased risk of GVHD, infection, renal, cardiovascular, and other complications, which can contribute to high mortality. Better understanding of molecular pathogenesis, improvement in posttransplant management, leading to early diagnosis, and management of TA-TMA are required to improve outcomes of this fatal entity.

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“…Проведение плазмообмена и назначение глюкокортикостероидов, успешно применяемых в терапии ТТП, при возник-новении ТА-ТМА не является оправданным -ответ на плазмаферез наблюдается менее чем в 50 % случа-ев, а летальность зачастую превышает 80 % [12]. В литературе описываются единичные случаи успеш-ного лечения ТА-ТМА винкристином, глюкокорти-костероидами, тиклопидина гидрохлоридом [13,14], экулизумабом [15] и ритуксимабом [12]. Общая вы-живаемость пациентов с/без ТА-ТМА представлена на рисунке.…”

Section: патогенез факторы риска и дифференциальная диагностикаunclassified

The case of successful therapy with rituximab secondary hemolytic-uremic syndrome in a patient after the second allogeneic hematopoietic stem cell transplantation

Пристанскова¹,

Kirgizov²,

Sidorova³

et al. 2016

Ross. ž. det. gematol. onkol.

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Recovery from life-threatening transplantation-associated thrombotic microangiopathy using eculizumab in a patient with very severe aplastic anemia

Cited by 17 publications

References 8 publications

Hematopoietic stem cell transplantation and acute kidney injury in children: A comprehensive review

Hematopoietic stem cell transplantation and acute kidney injury in children: A comprehensive review

Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy

The case of successful therapy with rituximab secondary hemolytic-uremic syndrome in a patient after the second allogeneic hematopoietic stem cell transplantation

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