Safety and efficacy of autologous stem cell transplantation in dialysis-dependent myeloma patients—The DIADEM study from the chronic malignancies working party of the EBMT

The role of high-dose chemotherapy followed by autologous stem cell transplantation (ASCT) in the treatment of myeloma (MM) patients with severe and/or dialysis-dependent renal impairment remains uncertain. We report on the outcomes of 110 patients (median age 57 years) who had become dialysis-depen...

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Veröffentlicht in:Bone marrow transplantation (Basingstoke) 2023-04, Vol.58 (4), p.424-429
Hauptverfasser: Waszczuk-Gajda, Anna, Gras, Luuk, de Wreede, Liesbeth C., Sirait, Tiarlan, Illes, Arpad, Ozkurt, Zubeyde Nur, Snowden, John A., Arat, Mutlu, Bulabois, Claude Eric, Niederland, Judith, Sever, Matjaz, Paneesha, Shankara, Potter, Victoria, Gadisseur, Alain, Chalopin, Thomas, Van Gorkom, Gwendolyn, López, Joaquin Martínez, Kerre, Tessa, Drozd-Sokolowska, Joanna, Raj, Kavita, Hayden, Patrick J., Beksac, Meral, Yakoub-Agha, Ibrahim, McLornan, Donal P., Schönland, Stefan
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container_end_page 429
container_issue 4
container_start_page 424
container_title Bone marrow transplantation (Basingstoke)
container_volume 58
creator Waszczuk-Gajda, Anna
Gras, Luuk
de Wreede, Liesbeth C.
Sirait, Tiarlan
Illes, Arpad
Ozkurt, Zubeyde Nur
Snowden, John A.
Arat, Mutlu
Bulabois, Claude Eric
Niederland, Judith
Sever, Matjaz
Paneesha, Shankara
Potter, Victoria
Gadisseur, Alain
Chalopin, Thomas
Van Gorkom, Gwendolyn
López, Joaquin Martínez
Kerre, Tessa
Drozd-Sokolowska, Joanna
Raj, Kavita
Hayden, Patrick J.
Beksac, Meral
Yakoub-Agha, Ibrahim
McLornan, Donal P.
Schönland, Stefan
description The role of high-dose chemotherapy followed by autologous stem cell transplantation (ASCT) in the treatment of myeloma (MM) patients with severe and/or dialysis-dependent renal impairment remains uncertain. We report on the outcomes of 110 patients (median age 57 years) who had become dialysis-dependent pre-ASCT and who underwent a first ASCT between 1997 and 2017. Sixty-three (57%) patients had light chain MM. All patients required dialysis (94% hemodialysis and 6% peritoneal). Forty-four of 71 (62%) patients received bortezomib-based induction regimens and 42 (39%) patients had achieved at least a very good partial response (VGPR) pre-ASCT. Melphalan dosing was as follows: ≤140 mg/m 2 (82%), and >140 mg/m 2 (18%). The median PFS after ASCT was 35 months (95% CI: 21.5–42.2) and the median OS 102 months (95% CI: 70.4–129.1). At 1, 2, and 5 years after ASCT, 8% (95% CI 3–14%), 13% (6–20%), and 20% (12–29%) of patients, respectively, had achieved dialysis independence. In multivariate analyses of OS and PFS including age at ASCT, response at ASCT, and year of ASCT, younger age at ASCT and better response at ASCT (CR/VGPR/PR vs. MR/SD/progression) were significantly associated with better OS and PFS.
doi_str_mv 10.1038/s41409-023-01915-7
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drug therapy</subject><subject>Myeloma</subject><subject>Public Health</subject><subject>Renal Dialysis</subject><subject>Renal function</subject><subject>Retrospective Studies</subject><subject>Stem Cell Transplantation</subject><subject>Stem Cells</subject><subject>Transplantation</subject><subject>Transplantation, Autologous</subject><subject>Treatment Outcome</subject><issn>0268-3369</issn><issn>1476-5365</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2023</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><sourceid>BENPR</sourceid><recordid>eNp9kc1u1DAUhSMEokPhBVggS2xgEfBfbGc5bQdaaSoWDGvLcZwZl8QebAeUHQ_Ba_BSPAlOU4rEgpXlez-fe31OUTxH8A2CRLyNFFFYlxCTEqIaVSV_UKwQ5aysCKseFiuImSgJYfVJ8STGGwgRpbB6XJwQxgTivFoVPz-qzqQJKNcC03VWKz0B3wE1Jt_7vR8jiMkMQJu-BykoF4-9ckkl6x2wDrRW9VO0sWzN0bjWuASGyfR-UOCYoXyPv77_2B0MuLhaX2yus9rYTqALfgApV_UheGc1GFRv9045bU0E33z4bN0-K4R0u81Mbs6ud0-LR53qo3l2d54Wn95tdueX5fbD-6vz9bbU2ZBUMtJ2GjUVw7DjDRWmabIJpqm1EBBrqOeWIrQWTAjKuDCQKtFwXGmMiOHktHi96B5UL4_BDipM0isrL9dbOdcgpaIWBH9FmX21sMfgv4wmJjnYONulnMn2ScyZwKSueZ3Rl_-gN34MLv8kU3UF6wpxnCm8UDr4GIPp7jdAUM7ByyV4mYOXt8HLeeMXd9JjM5j2_smfpDNAFiDmltub8Hf2f2R_Ax6Kuww</recordid><startdate>20230401</startdate><enddate>20230401</enddate><creator>Waszczuk-Gajda, Anna</creator><creator>Gras, Luuk</creator><creator>de Wreede, Liesbeth C.</creator><creator>Sirait, Tiarlan</creator><creator>Illes, Arpad</creator><creator>Ozkurt, Zubeyde Nur</creator><creator>Snowden, John A.</creator><creator>Arat, Mutlu</creator><creator>Bulabois, Claude Eric</creator><creator>Niederland, Judith</creator><creator>Sever, Matjaz</creator><creator>Paneesha, Shankara</creator><creator>Potter, Victoria</creator><creator>Gadisseur, Alain</creator><creator>Chalopin, Thomas</creator><creator>Van Gorkom, Gwendolyn</creator><creator>López, Joaquin Martínez</creator><creator>Kerre, Tessa</creator><creator>Drozd-Sokolowska, Joanna</creator><creator>Raj, Kavita</creator><creator>Hayden, Patrick J.</creator><creator>Beksac, Meral</creator><creator>Yakoub-Agha, Ibrahim</creator><creator>McLornan, Donal P.</creator><creator>Schönland, Stefan</creator><general>Nature Publishing Group UK</general><general>Nature Publishing Group</general><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>3V.</scope><scope>7QO</scope><scope>7QP</scope><scope>7T5</scope><scope>7U9</scope><scope>7X7</scope><scope>7XB</scope><scope>88E</scope><scope>8AO</scope><scope>8FD</scope><scope>8FE</scope><scope>8FH</scope><scope>8FI</scope><scope>8FJ</scope><scope>8FK</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>AZQEC</scope><scope>BBNVY</scope><scope>BENPR</scope><scope>BHPHI</scope><scope>CCPQU</scope><scope>DWQXO</scope><scope>FR3</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>GNUQQ</scope><scope>H94</scope><scope>HCIFZ</scope><scope>K9.</scope><scope>LK8</scope><scope>M0S</scope><scope>M1P</scope><scope>M7N</scope><scope>M7P</scope><scope>P64</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>7X8</scope><scope>1XC</scope><orcidid>https://orcid.org/0000-0003-1374-4503</orcidid><orcidid>https://orcid.org/0000-0002-4853-5579</orcidid><orcidid>https://orcid.org/0000-0002-8258-354X</orcidid><orcidid>https://orcid.org/0000-0003-2039-8557</orcidid><orcidid>https://orcid.org/0000-0003-4524-8782</orcidid><orcidid>https://orcid.org/0000-0003-1224-091X</orcidid><orcidid>https://orcid.org/0000-0001-6819-3476</orcidid><orcidid>https://orcid.org/0000-0002-4562-6264</orcidid><orcidid>https://orcid.org/0000-0002-7667-9369</orcidid></search><sort><creationdate>20230401</creationdate><title>Safety and efficacy of autologous stem cell transplantation in dialysis-dependent myeloma patients—The DIADEM study from the chronic malignancies working party of the EBMT</title><author>Waszczuk-Gajda, Anna ; Gras, Luuk ; de Wreede, Liesbeth C. ; Sirait, Tiarlan ; Illes, Arpad ; Ozkurt, Zubeyde Nur ; Snowden, John A. ; Arat, Mutlu ; Bulabois, Claude Eric ; Niederland, Judith ; Sever, Matjaz ; Paneesha, Shankara ; Potter, Victoria ; Gadisseur, Alain ; Chalopin, Thomas ; Van Gorkom, Gwendolyn ; López, Joaquin Martínez ; Kerre, Tessa ; Drozd-Sokolowska, Joanna ; Raj, Kavita ; Hayden, Patrick J. ; Beksac, Meral ; Yakoub-Agha, Ibrahim ; McLornan, Donal P. ; Schönland, Stefan</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c409t-63dfc1b5620f7b48ebb369eb9c8802c0cb562a34986884678e04a8b725c213e73</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2023</creationdate><topic>692/699/1541/1990/804</topic><topic>692/700/565/2319</topic><topic>Age</topic><topic>Antineoplastic Combined Chemotherapy Protocols - 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Academic</collection><collection>Hyper Article en Ligne (HAL)</collection><jtitle>Bone marrow transplantation (Basingstoke)</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Waszczuk-Gajda, Anna</au><au>Gras, Luuk</au><au>de Wreede, Liesbeth C.</au><au>Sirait, Tiarlan</au><au>Illes, Arpad</au><au>Ozkurt, Zubeyde Nur</au><au>Snowden, John A.</au><au>Arat, Mutlu</au><au>Bulabois, Claude Eric</au><au>Niederland, Judith</au><au>Sever, Matjaz</au><au>Paneesha, Shankara</au><au>Potter, Victoria</au><au>Gadisseur, Alain</au><au>Chalopin, Thomas</au><au>Van Gorkom, Gwendolyn</au><au>López, Joaquin Martínez</au><au>Kerre, Tessa</au><au>Drozd-Sokolowska, Joanna</au><au>Raj, Kavita</au><au>Hayden, Patrick J.</au><au>Beksac, Meral</au><au>Yakoub-Agha, Ibrahim</au><au>McLornan, Donal P.</au><au>Schönland, Stefan</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Safety and efficacy of autologous stem cell transplantation in dialysis-dependent myeloma patients—The DIADEM study from the chronic malignancies working party of the EBMT</atitle><jtitle>Bone marrow transplantation (Basingstoke)</jtitle><stitle>Bone Marrow Transplant</stitle><addtitle>Bone Marrow Transplant</addtitle><date>2023-04-01</date><risdate>2023</risdate><volume>58</volume><issue>4</issue><spage>424</spage><epage>429</epage><pages>424-429</pages><issn>0268-3369</issn><eissn>1476-5365</eissn><abstract>The role of high-dose chemotherapy followed by autologous stem cell transplantation (ASCT) in the treatment of myeloma (MM) patients with severe and/or dialysis-dependent renal impairment remains uncertain. We report on the outcomes of 110 patients (median age 57 years) who had become dialysis-dependent pre-ASCT and who underwent a first ASCT between 1997 and 2017. Sixty-three (57%) patients had light chain MM. All patients required dialysis (94% hemodialysis and 6% peritoneal). Forty-four of 71 (62%) patients received bortezomib-based induction regimens and 42 (39%) patients had achieved at least a very good partial response (VGPR) pre-ASCT. Melphalan dosing was as follows: ≤140 mg/m 2 (82%), and &gt;140 mg/m 2 (18%). The median PFS after ASCT was 35 months (95% CI: 21.5–42.2) and the median OS 102 months (95% CI: 70.4–129.1). At 1, 2, and 5 years after ASCT, 8% (95% CI 3–14%), 13% (6–20%), and 20% (12–29%) of patients, respectively, had achieved dialysis independence. In multivariate analyses of OS and PFS including age at ASCT, response at ASCT, and year of ASCT, younger age at ASCT and better response at ASCT (CR/VGPR/PR vs. MR/SD/progression) were significantly associated with better OS and PFS.</abstract><cop>London</cop><pub>Nature Publishing Group UK</pub><pmid>36681775</pmid><doi>10.1038/s41409-023-01915-7</doi><tpages>6</tpages><orcidid>https://orcid.org/0000-0003-1374-4503</orcidid><orcidid>https://orcid.org/0000-0002-4853-5579</orcidid><orcidid>https://orcid.org/0000-0002-8258-354X</orcidid><orcidid>https://orcid.org/0000-0003-2039-8557</orcidid><orcidid>https://orcid.org/0000-0003-4524-8782</orcidid><orcidid>https://orcid.org/0000-0003-1224-091X</orcidid><orcidid>https://orcid.org/0000-0001-6819-3476</orcidid><orcidid>https://orcid.org/0000-0002-4562-6264</orcidid><orcidid>https://orcid.org/0000-0002-7667-9369</orcidid></addata></record>
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identifier ISSN: 0268-3369
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issn 0268-3369
1476-5365
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subjects 692/699/1541/1990/804
692/700/565/2319
Age
Antineoplastic Combined Chemotherapy Protocols - therapeutic use
Autografts
Bortezomib
Bortezomib - therapeutic use
Cell Biology
Chemotherapy
Dialysis
Dosage
Hematology
Hematopoietic Stem Cell Transplantation
Hemodialysis
Humans
Internal Medicine
Life Sciences
Malignancy
Medicine
Medicine & Public Health
Melphalan
Middle Aged
Multiple myeloma
Multiple Myeloma - drug therapy
Myeloma
Public Health
Renal Dialysis
Renal function
Retrospective Studies
Stem Cell Transplantation
Stem Cells
Transplantation
Transplantation, Autologous
Treatment Outcome
title Safety and efficacy of autologous stem cell transplantation in dialysis-dependent myeloma patients—The DIADEM study from the chronic malignancies working party of the EBMT
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