Moxetumomab pasudotox in relapsed/refractory hairy cell leukemia
Robert Kreitman
(1)
,
Claire Dearden
(2)
,
Pier Luigi Zinzani
(3)
,
Julio Delgado
(4)
,
Lionel Karlin
(5)
,
Tadeusz Robak
(6)
,
Douglas Gladstone
(7)
,
Philipp Le Coutre
(8)
,
Sascha Dietrich
(9)
,
Mirjana Gotic
(10)
,
Loree Larratt
(11)
,
Fritz Offner
(12)
,
Gary Schiller
(13)
,
Ronan Swords
(14)
,
Larry Bacon
(15)
,
Monica Bocchia
(16)
,
Krimo Bouabdallah
(17)
,
Dimitri Breems
(18)
,
Agostino Cortelezzi
(19)
,
Shira Dinner
(20)
,
Michael Doubek
(21)
,
Bjørn Tore Gjertsen
(22)
,
Marco Gobbi
(23)
,
Andrzej Hellmann
(24)
,
Stéphane Leprêtre
(25, 26)
,
Frédéric Maloisel
(27)
,
Farhad Ravandi
(28)
,
Philipp Rousselot
(29)
,
Mathias Rummel
(30)
,
Tanya Siddiqi
(31)
,
Tamar Tadmor
(32)
,
Xavier Troussard
(33)
,
Cecilia Arana Yi
(34)
,
Giuseppe Saglio
(35)
,
Gail Roboz
(36)
,
Kemal Balic
(37)
,
Nathan Standifer
(37)
,
Peng He
(37)
,
Shannon Marshall
(37)
,
Wyndham Wilson
(38)
,
Ira Pastan
(38)
,
Nai-Shun Yao
(37)
,
Francis Giles
(20)
1
NCI-NIH -
National Cancer Institute [Bethesda]
2 Royal Marsden NHS Foundation Trust
3 UNIBO - Alma Mater Studiorum Università di Bologna = University of Bologna
4 CRESIB - Barcelona Centre for International Health Research, Hospital Clinic
5 Service d’Hématologie [Centre Hospitalier Lyon Sud - HCL]
6 MUL - Medical University of Łódź
7 JHU - Johns Hopkins University
8 Charité - UniversitätsMedizin = Charité - University Hospital [Berlin]
9 Universität Heidelberg [Heidelberg] = Heidelberg University
10 KCS - Clinical Center of Serbia
11 University of Alberta
12 UGENT - Universiteit Gent = Ghent University
13 David Geffen School of Medicine [Los Angeles]
14 University of Miami [Coral Gables]
15 St James's University Hospital
16 UNISI - Università degli Studi di Siena = University of Siena
17 CHU Bordeaux [Bordeaux]
18 ZNA - Ziekenhuis Netwerk Antwerpen
19 UNIMI - Università degli Studi di Milano = University of Milan
20 Northwestern University Feinberg School of Medicine
21 SCI / MUNI - Faculty of Science [Brno]
22 Haukeland University Hospital
23 Ospedale Policlinico San Martino [Genoa]
24 Medical University of Gdańsk
25 GPMCND - Génomique et Médecine Personnalisée du Cancer et des Maladies Neuropsychiatriques
26 CLCC Henri Becquerel - Centre de Lutte Contre le Cancer Henri Becquerel Normandie Rouen
27 Clinique Sainte Anne [Strasbourg]
28 MD Anderson Cancer Center [Houston]
29 2I - Infection et inflammation
30 JLU - Justus-Liebig-Universität Gießen = Justus Liebig University
31 City of Hope National Medical Center
32 Bnai Zion Medical Center [Israël]
33 Hôpital Côte de Nacre [CHU Caen]
34 The University of New Mexico [Albuquerque]
35 UNITO - Università degli studi di Torino = University of Turin
36 New York Presbyterian Hospital
37 MedImmune
38 NIH - National Institutes of Health [Bethesda]
2 Royal Marsden NHS Foundation Trust
3 UNIBO - Alma Mater Studiorum Università di Bologna = University of Bologna
4 CRESIB - Barcelona Centre for International Health Research, Hospital Clinic
5 Service d’Hématologie [Centre Hospitalier Lyon Sud - HCL]
6 MUL - Medical University of Łódź
7 JHU - Johns Hopkins University
8 Charité - UniversitätsMedizin = Charité - University Hospital [Berlin]
9 Universität Heidelberg [Heidelberg] = Heidelberg University
10 KCS - Clinical Center of Serbia
11 University of Alberta
12 UGENT - Universiteit Gent = Ghent University
13 David Geffen School of Medicine [Los Angeles]
14 University of Miami [Coral Gables]
15 St James's University Hospital
16 UNISI - Università degli Studi di Siena = University of Siena
17 CHU Bordeaux [Bordeaux]
18 ZNA - Ziekenhuis Netwerk Antwerpen
19 UNIMI - Università degli Studi di Milano = University of Milan
20 Northwestern University Feinberg School of Medicine
21 SCI / MUNI - Faculty of Science [Brno]
22 Haukeland University Hospital
23 Ospedale Policlinico San Martino [Genoa]
24 Medical University of Gdańsk
25 GPMCND - Génomique et Médecine Personnalisée du Cancer et des Maladies Neuropsychiatriques
26 CLCC Henri Becquerel - Centre de Lutte Contre le Cancer Henri Becquerel Normandie Rouen
27 Clinique Sainte Anne [Strasbourg]
28 MD Anderson Cancer Center [Houston]
29 2I - Infection et inflammation
30 JLU - Justus-Liebig-Universität Gießen = Justus Liebig University
31 City of Hope National Medical Center
32 Bnai Zion Medical Center [Israël]
33 Hôpital Côte de Nacre [CHU Caen]
34 The University of New Mexico [Albuquerque]
35 UNITO - Università degli studi di Torino = University of Turin
36 New York Presbyterian Hospital
37 MedImmune
38 NIH - National Institutes of Health [Bethesda]
Abstract
This is a pivotal, multicenter, open-label study of moxetumomab pasudotox, a recombinant CD22-targeting immunotoxin, in hairy cell leukemia (HCL), a rare B cell malignancy with high CD22 expression. The study enrolled patients with relapsed/ refractory HCL who had ≥2 prior systemic therapies, including ≥1 purine nucleoside analog. Patients received moxetumomab pasudotox 40 µg/kg intravenously on days 1, 3, and 5 every 28 days for ≤6 cycles. Blinded independent central review determined disease response and minimal residual disease (MRD) status. Among 80 patients (79% males; median age, 60.0 years), durable complete response (CR) rate was 30%, CR rate was 41%, and objective response rate (CR and partial response) was 75%; 64 patients (80%) achieved hematologic remission. Among complete responders, 27 (85%) achieved MRD negativity by immunohistochemistry. The most frequent adverse events (AEs) were peripheral edema (39%), nausea (35%), fatigue (34%), and headache (33%). Treatment-related serious AEs of hemolytic uremic syndrome (7.5%) and capillary leak syndrome (5%) were reversible and generally manageable with supportive care and treatment discontinuation (6 patients; 7.5%). Moxetumomab pasudotox treatment achieved a high rate of independently assessed durable response and MRD eradication in heavily pretreated patients with HCL, with acceptable tolerability.
Origin : Publication funded by an institution
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