Moxetumomab pasudotox in relapsed/refractory hairy cell
leukemia

J 2018

Moxetumomab pasudotox in relapsed/refractory hairy cell leukemia

KREITMAN, R.J., C. DEARDEN, P.L. ZINZANI, J. DELGADO, L. KARLIN et. al.

Basic information

Original name

Moxetumomab pasudotox in relapsed/refractory hairy cell leukemia

Authors

KREITMAN, R.J. (840 United States of America), C. DEARDEN (826 United Kingdom of Great Britain and Northern Ireland), P.L. ZINZANI (380 Italy), J. DELGADO (724 Spain), L. KARLIN (250 France), T. ROBAK (616 Poland), D.E. GLADSTONE (840 United States of America), P. LE COUTRE (276 Germany), S. DIETRICH (276 Germany), M. GOTIC (688 Serbia), L. LARRATT (124 Canada), F. OFFNER (56 Belgium), G. SCHILLER (840 United States of America), R. SWORDS (840 United States of America), L. BACON (372 Ireland), M. BOCCHIA (380 Italy), K. BOUABDALLAH (250 France), D.A. BREEMS (56 Belgium), A. CORTELEZZI (380 Italy), S. DINNER (840 United States of America), Michael DOUBEK (203 Czech Republic, belonging to the institution), B.T. GJERTSEN (578 Norway), M. GOBBI (380 Italy), A. HELLMANN (616 Poland), S. LEPRETRE (250 France), F. MALOISEL (250 France), F. RAVANDI (840 United States of America), P. ROUSSELOT (250 France), M. RUMMEL (276 Germany), T. SIDDIQI (840 United States of America), T. TADMOR (376 Israel), X. TROUSSARD (250 France), C.A. YI (250 France), G. SAGLIO (380 Italy), G.J. ROBOZ (840 United States of America), K. BALIC (840 United States of America), N. STANDIFER (840 United States of America), P. HE (840 United States of America), S. MARSHALL (840 United States of America), W. WILSON (840 United States of America), I. PASTAN (840 United States of America), N.S. YAO (840 United States of America) and F. GILES (840 United States of America)

Edition

Leukemia, London, Nature Publishing Group, 2018, 0887-6924

Other information

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

30205 Hematology

Country of publisher

United Kingdom of Great Britain and Northern Ireland

Confidentiality degree

není předmětem státního či obchodního tajemství

References:

URL

Impact factor

Impact factor: 9.944

RIV identification code

RIV/00216224:14110/18:00103867

Organization unit

Faculty of Medicine

DOI

http://dx.doi.org/10.1038/s41375-018-0210-1

UT WoS

000441284000009

Keywords in English

MINIMAL RESIDUAL DISEASE; TERM-FOLLOW-UP; CLADRIBINE; RITUXIMAB; IMMUNOHISTOCHEMISTRY; ERADICATION; DIAGNOSIS; EFFICACY; PATTERNS; ANTIBODY

Abstract

V originále

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 mu g/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.

Citovat

KREITMAN, R.J., C. DEARDEN, P.L. ZINZANI, J. DELGADO, L. KARLIN, T. ROBAK, D.E. GLADSTONE, P. LE COUTRE, S. DIETRICH, M. GOTIC, L. LARRATT, F. OFFNER, G. SCHILLER, R. SWORDS, L. BACON, M. BOCCHIA, K. BOUABDALLAH, D.A. BREEMS, A. CORTELEZZI, S. DINNER, Michael DOUBEK, B.T. GJERTSEN, M. GOBBI, A. HELLMANN, S. LEPRETRE, F. MALOISEL, F. RAVANDI, P. ROUSSELOT, M. RUMMEL, T. SIDDIQI, T. TADMOR, X. TROUSSARD, C.A. YI, G. SAGLIO, G.J. ROBOZ, K. BALIC, N. STANDIFER, P. HE, S. MARSHALL, W. WILSON, I. PASTAN, N.S. YAO and F. GILES. Moxetumomab pasudotox in relapsed/refractory hairy cell leukemia. Leukemia. London: Nature Publishing Group, 2018, vol. 32, No 8, p. 1768-1777. ISSN 0887-6924. Available from: https://dx.doi.org/10.1038/s41375-018-0210-1.

@article{1443161,
   author = {Kreitman, R.J. and Dearden, C. and Zinzani, P.L. and Delgado, J. and Karlin, L. and Robak, T. and Gladstone, D.E. and le Coutre, P. and Dietrich, S. and Gotic, M. and Larratt, L. and Offner, F. and Schiller, G. and Swords, R. and Bacon, L. and Bocchia, M. and Bouabdallah, K. and Breems, D.A. and Cortelezzi, A. and Dinner, S. and Doubek, Michael and Gjertsen, B.T. and Gobbi, M. and Hellmann, A. and Lepretre, S. and Maloisel, F. and Ravandi, F. and Rousselot, P. and Rummel, M. and Siddiqi, T. and Tadmor, T. and Troussard, X. and Yi, C.A. and Saglio, G. and Roboz, G.J. and Balic, K. and Standifer, N. and He, P. and Marshall, S. and Wilson, W. and Pastan, I. and Yao, N.S. and Giles, F.},
   article_location = {London},
   article_number = {8},
   doi = {http://dx.doi.org/10.1038/s41375-018-0210-1},
   keywords = {MINIMAL RESIDUAL DISEASE; TERM-FOLLOW-UP; CLADRIBINE; RITUXIMAB; IMMUNOHISTOCHEMISTRY; ERADICATION; DIAGNOSIS; EFFICACY; PATTERNS; ANTIBODY},
   language = {eng},
   issn = {0887-6924},
   journal = {Leukemia},
   title = {Moxetumomab pasudotox in relapsed/refractory hairy cell leukemia},
   url = {https://www.nature.com/articles/s41375-018-0210-1.pdf},
   volume = {32},
   year = {2018}
}

TY  - JOUR
ID  - 1443161
AU  - Kreitman, R.J. - Dearden, C. - Zinzani, P.L. - Delgado, J. - Karlin, L. - Robak, T. - Gladstone, D.E. - le Coutre, P. - Dietrich, S. - Gotic, M. - Larratt, L. - Offner, F. - Schiller, G. - Swords, R. - Bacon, L. - Bocchia, M. - Bouabdallah, K. - Breems, D.A. - Cortelezzi, A. - Dinner, S. - Doubek, Michael - Gjertsen, B.T. - Gobbi, M. - Hellmann, A. - Lepretre, S. - Maloisel, F. - Ravandi, F. - Rousselot, P. - Rummel, M. - Siddiqi, T. - Tadmor, T. - Troussard, X. - Yi, C.A. - Saglio, G. - Roboz, G.J. - Balic, K. - Standifer, N. - He, P. - Marshall, S. - Wilson, W. - Pastan, I. - Yao, N.S. - Giles, F.
PY  - 2018
TI  - Moxetumomab pasudotox in relapsed/refractory hairy cell leukemia
JF  - Leukemia
VL  - 32
IS  - 8
SP  - 1768-1777
EP  - 1768-1777
PB  - Nature Publishing Group
SN  - 08876924
KW  - MINIMAL RESIDUAL DISEASE
KW  - TERM-FOLLOW-UP
KW  - CLADRIBINE
KW  - RITUXIMAB
KW  - IMMUNOHISTOCHEMISTRY
KW  - ERADICATION
KW  - DIAGNOSIS
KW  - EFFICACY
KW  - PATTERNS
KW  - ANTIBODY
UR  - https://www.nature.com/articles/s41375-018-0210-1.pdf
N2  - 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 mu g/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.
ER  -

KREITMAN, R.J., C. DEARDEN, P.L. ZINZANI, J. DELGADO, L. KARLIN, T. ROBAK, D.E. GLADSTONE, P. LE COUTRE, S. DIETRICH, M. GOTIC, L. LARRATT, F. OFFNER, G. SCHILLER, R. SWORDS, L. BACON, M. BOCCHIA, K. BOUABDALLAH, D.A. BREEMS, A. CORTELEZZI, S. DINNER, Michael DOUBEK, B.T. GJERTSEN, M. GOBBI, A. HELLMANN, S. LEPRETRE, F. MALOISEL, F. RAVANDI, P. ROUSSELOT, M. RUMMEL, T. SIDDIQI, T. TADMOR, X. TROUSSARD, C.A. YI, G. SAGLIO, G.J. ROBOZ, K. BALIC, N. STANDIFER, P. HE, S. MARSHALL, W. WILSON, I. PASTAN, N.S. YAO and F. GILES. Moxetumomab pasudotox in relapsed/refractory hairy cell leukemia. \textit{Leukemia}. London: Nature Publishing Group, 2018, vol.~32, No~8, p.~1768-1777. ISSN~0887-6924. Available from: https://dx.doi.org/10.1038/s41375-018-0210-1.

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