Diagnosis and management of Waldenström macroglobulinemia: Mayo Stratification of Macroglobulinemia and Risk-Adapted Therapy (mSMART) guidelines 2016

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Abstract

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.

Original languageEnglish (US)
Pages (from-to)1257-1265
Number of pages9
JournalJAMA oncology
Volume3
Issue number9
DOIs
StatePublished - Sep 1 2017

Fingerprint

Waldenstrom Macroglobulinemia
Guidelines
Stem Cell Transplantation
Therapeutics
Clinical Trials
Recurrence
Proteasome Inhibitors
Plasma Exchange
Retreatment
Hematologic Diseases
Alkylating Agents
Sirolimus
Cyclophosphamide
Dexamethasone
Reaction Time
Immunoglobulin M
Lymphoma
Consensus
Stem Cells
Randomized Controlled Trials

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

Cite this

@article{4f256f1d4ab74139bd29380ba682c573,
title = "Diagnosis and management of Waldenstr{\"o}m macroglobulinemia: Mayo Stratification of Macroglobulinemia and Risk-Adapted Therapy (mSMART) guidelines 2016",
abstract = "IMPORTANCE: Waldenstr{\"o}m 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: Waldenstr{\"o}m 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.",
author = "Prashant Kapoor and Ansell, {Stephen Maxted} and Rafael Fonseca and {Chanan Khan}, {Asher A} and Kyle, {Robert A.} and Kumar, {Shaji K} and Mikhael, {Joseph R} and Witzig, {Thomas Elmer} and Mauermann, {Michelle M} and Angela Dispenzieri and Sikander Ailawadhi and Stewart, {Alexander Keith} and Martha Lacy and Thompson, {Carrie A} and Buadi, {Francis K.} and Dingli, {David M} and Morice, {William G.} and Go, {Ronald S.} and Dragan Jevremovic and Taimur Sher and Rebecca King and Braggio, {Esteban D} and Novak, {Anne J} and Vivek Roy and Ketterling, {Rhett P.} and Greipp, {Patricia T} and Martha Grogan and Ivana Micallef and Bergsagel, {Peter Leif} and Colgan, {Joseph P.} and Nelson Leung and Wilson Gonsalves and Yi Lin and Inwards, {David J.} and Hayman, {Suzanne R.} and Nowakowski, {Grzegorz S} and Johnston, {Patrick Bruce} and Russell, {Stephen J} and Markovic, {Svetomir Nenad} and Zeldenrust, {Steven R.} and Hwa, {Yi L.} and Lust, {John A.} and Porrata, {Luis F.} and Habermann, {Thomas Matthew} and Rajkumar, {S Vincent} and Morie Gertz and Reeder, {Craig B.}",
year = "2017",
month = "9",
day = "1",
doi = "10.1001/jamaoncol.2016.5763",
language = "English (US)",
volume = "3",
pages = "1257--1265",
journal = "JAMA oncology",
issn = "2374-2437",
publisher = "American Medical Association",
number = "9",

}

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 Maxted

AU - Fonseca, Rafael

AU - Chanan Khan, Asher A

AU - Kyle, Robert A.

AU - Kumar, Shaji K

AU - Mikhael, Joseph R

AU - Witzig, Thomas Elmer

AU - Mauermann, Michelle M

AU - Dispenzieri, Angela

AU - Ailawadhi, Sikander

AU - Stewart, Alexander Keith

AU - Lacy, Martha

AU - Thompson, Carrie A

AU - Buadi, Francis K.

AU - Dingli, David M

AU - Morice, William G.

AU - Go, Ronald S.

AU - Jevremovic, Dragan

AU - Sher, Taimur

AU - King, Rebecca

AU - Braggio, Esteban D

AU - Novak, Anne J

AU - Roy, Vivek

AU - Ketterling, Rhett P.

AU - Greipp, Patricia T

AU - Grogan, Martha

AU - Micallef, Ivana

AU - Bergsagel, Peter Leif

AU - Colgan, Joseph P.

AU - Leung, Nelson

AU - Gonsalves, Wilson

AU - Lin, Yi

AU - Inwards, David J.

AU - Hayman, Suzanne R.

AU - Nowakowski, Grzegorz S

AU - Johnston, Patrick Bruce

AU - Russell, Stephen J

AU - Markovic, Svetomir Nenad

AU - Zeldenrust, Steven R.

AU - Hwa, Yi L.

AU - Lust, John A.

AU - Porrata, Luis F.

AU - Habermann, Thomas Matthew

AU - Rajkumar, S Vincent

AU - Gertz, Morie

AU - Reeder, Craig B.

PY - 2017/9/1

Y1 - 2017/9/1

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.

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