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Long-term eculizumab treatment of aHUS patients with a long disease duration and chronic kidney disease: efficacy and safety outcomes

Questo Abstract è stato accettato come Comunicazione.

Razionale

Eculizumab (Ecu), a terminal complement inhibitor, is the first approved treatment for aHUS.  We report long-term follow-up data from a phase 2 trial in which pts with aHUS with a long disease duration and CKD were treated with Ecu.

Casistica e Metodi

Pts aged ≥12 yrs with aHUS and receiving chronic plasma exchange/plasma infusion (PE/PI) on a stable regimen were enrolled in an open-label, single-arm, phase 2 trial. PE/PI was discontinued and Ecu initiated (900 mg/wk for 4 wks, 1200 mg at wk 5, then 1200 mg q2 wks thereafter). Primary endpoints (26wks) were TMA event-free status (≥12 consecutive wks with no platelet count change >25% + no PE/PI + no new dialysis) and haematologic normalisation (platelet count ≥150´109/L and lactate dehydrogenase ≤ upper limit of normal sustained ≥4 weeks). Data were also collected up to 2 yrs of treatment in the extension study.

Risultati

20 pts were enrolled in the 26-wk trial; 19 pts entered the long-term extension. TMA event‑free status was achieved in 16 pts (80%) at 26 wks and in 19 pts (95%) at a median follow-up of 114 wks. Haematologic normalisation was achieved in 18 pts (90%) by 26 wks and was maintained at a median of 114 weeks. At 26 wks haemoglobin (Hb) level and eGFR were significantly improved, and continued to improve during the extension period (Table). Mean platelet count ±SD was 228±77.66x109/L at baseline and did not significantly change during treatment. Ecu was well tolerated and AE rates remained steady or declined with longer duration of Ecu treatment.

Conclusioni

Long-term treatment with Ecu in aHUS patients with long disease duration was associated with a sustained suppression of TMA, continued and significant improvement in renal function and was well tolerated. These results support the importance of ongoing Ecu treatment in these aHUS pts.

Trivelli A1, Licht C2, Legendre C3, Douglas K4, Herthelius M5, Goodship T6, Remuzzi G7, Bedrosian CL8, Loirat C9
(1Istituto G Gaslini, Genoa, Italy , 2Hospital for Sick Children, Toronto, ON, Canada; 3Hôpital Necker, Paris, France; 4Beatson West Scotland Cancer Centre, Glasgow, UK; 5Karolinska University Hospital, Stockholm, Sweden; 6Newcastle Univ., Newcastle, UK; 7Mario Negri Institute for Pharmacological Research IRCCS, Bergamo, Italy; 8Alexion Pharmaceuticals Inc., Cheshire, CT, USA; 9Hôpital Debré, Paris, France )
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Realizzazione: Tesi S.p.A.

Per assistenza contattare: Lucia Piumetto, Tesi S.p.A.
0172 476301 — lucia.piumetto@gruppotesi.com