Use of Eculizumab in Patients With Allogeneic Stem Cell Transplant-Associated Thrombotic Microangiopathy

Fontbrune, Flore Sicre de; Garrigue, Claire; Sîrvent, Anne; Huynh, Anne; Faguer, Stanislas; Nguyen, Stephanie; Bay, Jacques‐Olivier; Neven, Bénédicte; Moussi, J F.; Simon, Laurence; Xhaard, Aliénor; Resche-Riggon, Matthieu; O'Meara, Alix; Frémeaux‐Bacchi, Véronique; Veyradier, Agnès; Socié, Gérard; Coppo, Paul; Latour, R.P. de

doi:10.1097/tp.0000000000000601

Cited by 118 publications

(107 citation statements)

References 18 publications

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“…63,68 Eculizumab has been shown to reverse end-organ damage and restore hematologic parameters in children and adults, without a higher incidence of bacterial infections. 69 . 70,71 Dakhal et al 72 published the results of a MEDLINE search of all case reports of TA-TMA and solid organ transplantation-associated TMA treated with eculizumab until November of 2014.…”

Section: Treatmentmentioning

confidence: 99%

None of the above: thrombotic microangiopathy beyond TTP and HUS

Masias

Vasu

Cataland

2017

Blood

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Acquired thrombotic thrombocytopenic purpura (TTP) and hemolytic uremic syndrome (HUS) are appropriately at the top of a clinician’s differential when a patient presents with a clinical picture consistent with an acute thrombotic microangiopathy (TMA). However, there are several additional diagnoses that should be considered in patients presenting with an acute TMA, especially in patients with nondeficient ADAMTS13 (a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13) activity (>10%). An increased awareness of drug-induced TMA is also essential because the key to their diagnosis more often is an appropriately detailed medical history to inquire about potential exposures. Widespread inflammation and endothelial damage are central in the pathogenesis of the TMA, with the treatment directed at the underlying disease if possible. TMA presentations in the critically ill, drug-induced TMA, cancer-associated TMA, and hematopoietic transplant–associated TMA (TA-TMA) and their specific treatment, where applicable, will be discussed in this manuscript. A complete assessment of all the potential etiologies for the TMA findings including acquired TTP will allow for a more accurate diagnosis and prevent prolonged or inappropriate treatment with plasma exchange therapy when it is less likely to be successful.

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Section: Treatmentmentioning

confidence: 99%

None of the above: thrombotic microangiopathy beyond TTP and HUS

Masias

Vasu

Cataland

2017

Blood

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“…Eculizumab has been studied as a potential therapeutic intervention for post-HSCT TMA, with outcomes that are significantly superior to prior cohorts of patients who did not receive the drug [87]. As this is a very expensive drug, strategies to optimize dosing have been proposed, including targeted serum drug levels or complement activity monitoring (CH50) until such a time as a clinical response has been achieved [88].…”

Section: What Is the Role Of Routine Screening For Thrombotic Microanmentioning

confidence: 99%

Complex Transfusion Issues in Pediatric Hematopoietic Stem Cell Transplantation

Webb

Abraham

2016

Transfusion Medicine Reviews

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“…It has been not clarified so far whether atypical HUS after alloSCT requires lifelong therapy with eculizumab like patients suffering from PNH. In their series, de Fontbrune et al [7] terminated eculizumab only in nonresponders or in the case of other severe problems such as refractory GvHD or relapsing malignancy. Termination of eculizumab would be desirable after alloSCT with or without GvHD, since it leads to additional immunosuppression by blocking complement activation.…”

Section: Tablementioning

confidence: 99%

“…The major indication for eculizumab is paroxysmal nocturnal hemoglobinuria (PNH). However, it has been used with success in atypical hemolytic uremic syndrome (HUS), even after alloSCT [7,8]. Eculizumab has been recommended as a lifelong therapy since PNH clones will not be eliminated and genetic alterations leading to atypical HUS persist.…”

Section: Tablementioning

confidence: 99%