TY - JOUR
T1 - Diagnosis and management of Waldenström macroglobulinemia
T2 - Mayo Stratification of Macroglobulinemia and Risk-Adapted Therapy (mSMART) guidelines 2016
AU - Kapoor, Prashant
AU - Ansell, Stephen M.
AU - Fonseca, Rafael
AU - Chanan-Khan, Asher
AU - Kyle, Robert A.
AU - Kumar, Shaji K.
AU - Mikhael, Joseph R.
AU - Witzig, Thomas E.
AU - Mauermann, Michelle
AU - Dispenzieri, Angela
AU - Ailawadhi, Sikander
AU - Stewart, A. Keith
AU - Lacy, Martha Q.
AU - Thompson, Carrie A.
AU - Buadi, Francis K.
AU - Dingli, David
AU - Morice, William G.
AU - Go, Ronald S.
AU - Jevremovic, Dragan
AU - Sher, Taimur
AU - King, Rebecca L.
AU - Braggio, Esteban
AU - Novak, Ann
AU - Roy, Vivek
AU - Ketterling, Rhett P.
AU - Greipp, Patricia T.
AU - Grogan, Martha
AU - Micallef, Ivana N.
AU - Bergsagel, P. Leif
AU - Colgan, Joseph P.
AU - Leung, Nelson
AU - Gonsalves, Wilson I.
AU - Lin, Yi
AU - Inwards, David J.
AU - Hayman, Suzanne R.
AU - Nowakowski, Grzegorz S.
AU - Johnston, Patrick B.
AU - Russell, Steven J.
AU - Markovic, Svetomir N.
AU - Zeldenrust, Steven R.
AU - Hwa, Yi L.
AU - Lust, John A.
AU - Porrata, Luis F.
AU - Habermann, Thomas M.
AU - Rajkumar, S. Vincent
AU - Gertz, Morie A.
AU - Reeder, Craig B.
N1 - Publisher Copyright:
© 2017 American Medical Association. All rights reserved.
PY - 2017/9
Y1 - 2017/9
N2 - IMPORTANCE: Waldenström macroglobulinemia (WM), an IgM-associated lymphoplasmacytic lymphoma, has witnessed several practice-altering advances in recent years. With availability of a wider array of therapies, the management strategies have become increasingly complex. Our multidisciplinary team appraised studies published or presented up to December 2015 to provide consensus recommendations for a risk-adapted approach to WM, using a grading system. OBSERVATIONS: Waldenström macroglobulinemia remains a rare, incurable cancer, with a heterogeneous disease course. The major classes of effective agents in WM include monoclonal antibodies, alkylating agents, purine analogs, proteasome inhibitors, immunomodulatory drugs, and mammalian target of rapamycin inhibitors. However, the highest-quality evidence from rigorously conducted randomized clinical trials remains scant. CONCLUSIONS AND RELEVANCE: Recognizing the paucity of data, we advocate participation in clinical trials, if available, at every stage of WM. Specific indications exist for initiation of therapy. Outside clinical trials, based on the synthesis of available evidence, we recommend bendamustine-rituximab as primary therapy for bulky disease, profound hematologic compromise, or constitutional symptoms attributable to WM. Dexamethasone-rituximab-cyclophosphamide is an alternative, particularly for nonbulky WM. Routine rituximab maintenance should be avoided. Plasma exchange should be promptly initiated before cytoreduction for hyperviscosity-related symptoms. Stem cell harvest for future use may be considered in first remission for patients 70 years or younger who are potential candidates for autologous stem cell transplantation. At relapse, retreatment with the original therapy is reasonable in patients with prior durable responses (time to next therapy 3 years) and good tolerability to previous regimen. Ibrutinib is efficacious in patients with relapsed or refractory disease harboring MYD88 L265P mutation. In the absence of neuropathy, a bortezomib-rituximab–based option is reasonable for relapsed or refractory disease. In select patients with chemosensitive disease, autologous stem cell transplantation should be considered at first or second relapse. Everolimus and purine analogs are suitable options for refractory or multiply relapsed WM. Our recommendations are periodically updated as new, clinically relevant information emerges.
AB - IMPORTANCE: Waldenström macroglobulinemia (WM), an IgM-associated lymphoplasmacytic lymphoma, has witnessed several practice-altering advances in recent years. With availability of a wider array of therapies, the management strategies have become increasingly complex. Our multidisciplinary team appraised studies published or presented up to December 2015 to provide consensus recommendations for a risk-adapted approach to WM, using a grading system. OBSERVATIONS: Waldenström macroglobulinemia remains a rare, incurable cancer, with a heterogeneous disease course. The major classes of effective agents in WM include monoclonal antibodies, alkylating agents, purine analogs, proteasome inhibitors, immunomodulatory drugs, and mammalian target of rapamycin inhibitors. However, the highest-quality evidence from rigorously conducted randomized clinical trials remains scant. CONCLUSIONS AND RELEVANCE: Recognizing the paucity of data, we advocate participation in clinical trials, if available, at every stage of WM. Specific indications exist for initiation of therapy. Outside clinical trials, based on the synthesis of available evidence, we recommend bendamustine-rituximab as primary therapy for bulky disease, profound hematologic compromise, or constitutional symptoms attributable to WM. Dexamethasone-rituximab-cyclophosphamide is an alternative, particularly for nonbulky WM. Routine rituximab maintenance should be avoided. Plasma exchange should be promptly initiated before cytoreduction for hyperviscosity-related symptoms. Stem cell harvest for future use may be considered in first remission for patients 70 years or younger who are potential candidates for autologous stem cell transplantation. At relapse, retreatment with the original therapy is reasonable in patients with prior durable responses (time to next therapy 3 years) and good tolerability to previous regimen. Ibrutinib is efficacious in patients with relapsed or refractory disease harboring MYD88 L265P mutation. In the absence of neuropathy, a bortezomib-rituximab–based option is reasonable for relapsed or refractory disease. In select patients with chemosensitive disease, autologous stem cell transplantation should be considered at first or second relapse. Everolimus and purine analogs are suitable options for refractory or multiply relapsed WM. Our recommendations are periodically updated as new, clinically relevant information emerges.
UR - http://www.scopus.com/inward/record.url?scp=85019840163&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85019840163&partnerID=8YFLogxK
U2 - 10.1001/jamaoncol.2016.5763
DO - 10.1001/jamaoncol.2016.5763
M3 - Article
C2 - 28056114
AN - SCOPUS:85019840163
SN - 2374-2437
VL - 3
SP - 1257
EP - 1265
JO - JAMA Oncology
JF - JAMA Oncology
IS - 9
ER -