Outcomes after Umbilical Cord Blood Transplantation for Myelodysplastic Syndromes

Aaron T. Gerds; Kwang Woo Ahn; Zhen Huan Hu; Hisham Abdel-Azim; Gorgun Akpek; Mahmoud Aljurf; Karen K. Ballen; Amer Beitinjaneh; Ulrike Bacher; Jean Yves Cahn; Saurabh Chhabra; Corey Cutler; Andrew Daly; Zachariah DeFilipp; Robert Peter Gale; Usama Gergis; Michael R. Grunwald; Gregory A. Hale; Betty Ky Hamilton; Madan Jagasia; Rammurti T. Kamble; Tamila Kindwall-Keller; Taiga Nishihori; Richard F. Olsson; Muthalagu Ramanathan; Ayman A. Saad; Melhem Solh; Celalettin Ustun; David Valcárcel; Erica Warlick; Baldeep M. Wirk; Matt Kalaycio; Edwin Alyea; Uday Popat; Ronald Sobecks; Wael Saber

doi:10.1016/j.bbmt.2017.03.014

Outcomes after Umbilical Cord Blood Transplantation for Myelodysplastic Syndromes

Aaron T. Gerds, Kwang Woo Ahn, Zhen Huan Hu, Hisham Abdel-Azim, Gorgun Akpek, Mahmoud Aljurf, Karen K. Ballen, Amer Beitinjaneh, Ulrike Bacher, Jean Yves Cahn, Saurabh Chhabra, Corey Cutler, Andrew Daly, Zachariah DeFilipp, Robert Peter Gale, Usama Gergis, Michael R. Grunwald, Gregory A. Hale, Betty Ky Hamilton, Madan JagasiaRammurti T. Kamble, Tamila Kindwall-Keller, Taiga Nishihori, Richard F. Olsson, Muthalagu Ramanathan, Ayman A. Saad, Melhem Solh, Celalettin Ustun, David Valcárcel, Erica Warlick, Baldeep M. Wirk, Matt Kalaycio, Edwin Alyea, Uday Popat, Ronald Sobecks, Wael Saber

Medicine - Hematology, Oncology, Transplant

Research output: Contribution to journal › Article › peer-review

16 Scopus citations

Abstract

For patients with hematologic malignancies undergoing allogeneic hematopoietic cell transplantation, umbilical cord blood transplantation (UCBT) has become an acceptable alternative donor source in the absence of a matched sibling or unrelated donor. To date, however, there have been few published series dedicated solely to describing the outcomes of adult patients with myelodysplastic syndrome (MDS) who have undergone UCBT. Between 2004 and 2013, 176 adults with MDS underwent UCBT as reported to the Center for International Blood and Marrow Transplant Research. Median age at the time of transplantation was 56 years (range, 18-73 years). The study group included 10% with very low, 23% with low, 19% with intermediate, 19% with high, and 13% with very high-risk Revised International Prognostic Scoring System (IPSS-R) scores. The 100-day probability of grade II-IV acute graft-versus-host disease (GVHD) was 38%, and the 3-year probability of chronic GVHD was 28%. The probabilities of relapse and transplantation-related mortality (TRM) at 3 years were 32% and 40%, respectively, leading to a 3-year disease-free survival (DFS) of 28% and an overall survival (OS) of 31%. In multivariate analysis, increasing IPSS-R score at the time of HCT was associated with inferior TRM (P = .0056), DFS (P = .018), and OS (P = .0082), but not with GVHD or relapse. The presence of pretransplantation comorbidities was associated with TRM (P = .001), DFS (P = .02), and OS (P = .001). Reduced-intensity conditioning was associated with increased risk of relapse (relative risk, 3.95; 95% confidence interval, 1.78-8.75; P < .001), and although a higher proportion of myeloablative UCBTs were performed in patients with high-risk disease, the effect of conditioning regimen intensity was the same regardless of IPSS-R score. For some individuals who lack a matched sibling or unrelated donor, UCBT can result in long-term DFS; however, the success of UCBT in this population is hampered by a high rate of TRM.

Original language	English (US)
Pages (from-to)	971-979
Number of pages	9
Journal	Biology of Blood and Marrow Transplantation
Volume	23
Issue number	6
DOIs	https://doi.org/10.1016/j.bbmt.2017.03.014
State	Published - Jun 2017

Bibliographical note

Funding Information:
Financial disclosure: The CIBMTR is supported by Public Health Service Grant/Cooperative Agreement 5U24-CA076518 from the National Cancer Institute (NCI), the National Heart, Lung and Blood Institute (NHLBI), and the National Institute of Allergy and Infectious Diseases (NIAID); Grant/Cooperative Agreement 5U10HL069294 from the NHLBI and NCI; Contract HHSH250201200016C) with the Health Resources and Services Administration (HRSA/DHHS); Grants N00014-13-1-0039 and N00014-14-1-0028 from the Office of Naval Research; and grants from Alexion; *Amgen, Inc.; Anonymous donation to the Medical College of Wisconsin; Be the Match Foundation; *Bristol Myers Squibb Oncology; *Celgene Corporation; *Chimerix, Inc.; Fred Hutchinson Cancer Research Center; Gamida Cell Ltd.; Genentech, Inc.; Genzyme Corporation; *Gilead Sciences, Inc.; Health Research, Inc. Roswell Park Cancer Institute; HistoGenetics, Inc.; Incyte Corporation; *Jazz Pharmaceuticals, Inc.; Jeff Gordon Children's Foundation; The Leukemia & Lymphoma Society; The Medical College of Wisconsin; Merck & Co, Inc.; Mesoblast; *Millennium: The Takeda Oncology Co.; *Miltenyi Biotec, Inc.; National Marrow Donor Program; Neovii Biotech NA, Inc.; Novartis Pharmaceuticals Corporation; Onyx Pharmaceuticals; Optum Healthcare Solutions, Inc.; Otsuka America Pharmaceutical, Inc.; Otsuka Pharmaceutical Co, Ltd. – Japan; Oxford Immunotec; Perkin Elmer, Inc.; Pharmacyclics; *Sanofi US; Seattle Genetics; Sigma-Tau Pharmaceuticals; *Spectrum Pharmaceuticals, Inc.; St. Baldrick's Foundation; *Sunesis Pharmaceuticals, Inc.; Swedish Orphan Biovitrum, Inc.; Telomere Diagnostics, Inc.; TerumoBCT; Therakos, Inc.; University of Minnesota; and *Wellpoint, Inc. The views expressed in this article do not reflect the official policy or position of the National Institutes of Health, the Department of the Navy, the Department of Defense, the Health Resources and Services Administration, or any other agency of the US Government.

Publisher Copyright:
© 2017 The American Society for Blood and Marrow Transplantation

Keywords

Blood and marrow transplantation
Myelodysplastic syndrome
Umbilical cord transplantation

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10.1016/j.bbmt.2017.03.014

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5474679

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Gerds, A. T., Woo Ahn, K., Hu, Z. H., Abdel-Azim, H., Akpek, G., Aljurf, M., Ballen, K. K., Beitinjaneh, A., Bacher, U., Cahn, J. Y., Chhabra, S., Cutler, C., Daly, A., DeFilipp, Z., Gale, R. P., Gergis, U., Grunwald, M. R., Hale, G. A., Hamilton, B. K., ... Saber, W. (2017). Outcomes after Umbilical Cord Blood Transplantation for Myelodysplastic Syndromes. Biology of Blood and Marrow Transplantation, 23(6), 971-979. https://doi.org/10.1016/j.bbmt.2017.03.014

Gerds, AT, Woo Ahn, K, Hu, ZH, Abdel-Azim, H, Akpek, G, Aljurf, M, Ballen, KK, Beitinjaneh, A, Bacher, U, Cahn, JY, Chhabra, S, Cutler, C, Daly, A, DeFilipp, Z, Gale, RP, Gergis, U, Grunwald, MR, Hale, GA, Hamilton, BK, Jagasia, M, Kamble, RT, Kindwall-Keller, T, Nishihori, T, Olsson, RF, Ramanathan, M, Saad, AA, Solh, M, Ustun, C, Valcárcel, D, Warlick, E, Wirk, BM, Kalaycio, M, Alyea, E, Popat, U, Sobecks, R & Saber, W 2017, 'Outcomes after Umbilical Cord Blood Transplantation for Myelodysplastic Syndromes', Biology of Blood and Marrow Transplantation, vol. 23, no. 6, pp. 971-979. https://doi.org/10.1016/j.bbmt.2017.03.014

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title = "Outcomes after Umbilical Cord Blood Transplantation for Myelodysplastic Syndromes",

abstract = "For patients with hematologic malignancies undergoing allogeneic hematopoietic cell transplantation, umbilical cord blood transplantation (UCBT) has become an acceptable alternative donor source in the absence of a matched sibling or unrelated donor. To date, however, there have been few published series dedicated solely to describing the outcomes of adult patients with myelodysplastic syndrome (MDS) who have undergone UCBT. Between 2004 and 2013, 176 adults with MDS underwent UCBT as reported to the Center for International Blood and Marrow Transplant Research. Median age at the time of transplantation was 56 years (range, 18-73 years). The study group included 10% with very low, 23% with low, 19% with intermediate, 19% with high, and 13% with very high-risk Revised International Prognostic Scoring System (IPSS-R) scores. The 100-day probability of grade II-IV acute graft-versus-host disease (GVHD) was 38%, and the 3-year probability of chronic GVHD was 28%. The probabilities of relapse and transplantation-related mortality (TRM) at 3 years were 32% and 40%, respectively, leading to a 3-year disease-free survival (DFS) of 28% and an overall survival (OS) of 31%. In multivariate analysis, increasing IPSS-R score at the time of HCT was associated with inferior TRM (P = .0056), DFS (P = .018), and OS (P = .0082), but not with GVHD or relapse. The presence of pretransplantation comorbidities was associated with TRM (P = .001), DFS (P = .02), and OS (P = .001). Reduced-intensity conditioning was associated with increased risk of relapse (relative risk, 3.95; 95% confidence interval, 1.78-8.75; P < .001), and although a higher proportion of myeloablative UCBTs were performed in patients with high-risk disease, the effect of conditioning regimen intensity was the same regardless of IPSS-R score. For some individuals who lack a matched sibling or unrelated donor, UCBT can result in long-term DFS; however, the success of UCBT in this population is hampered by a high rate of TRM.",

keywords = "Blood and marrow transplantation, Myelodysplastic syndrome, Umbilical cord transplantation",

author = "Gerds, {Aaron T.} and {Woo Ahn}, Kwang and Hu, {Zhen Huan} and Hisham Abdel-Azim and Gorgun Akpek and Mahmoud Aljurf and Ballen, {Karen K.} and Amer Beitinjaneh and Ulrike Bacher and Cahn, {Jean Yves} and Saurabh Chhabra and Corey Cutler and Andrew Daly and Zachariah DeFilipp and Gale, {Robert Peter} and Usama Gergis and Grunwald, {Michael R.} and Hale, {Gregory A.} and Hamilton, {Betty Ky} and Madan Jagasia and Kamble, {Rammurti T.} and Tamila Kindwall-Keller and Taiga Nishihori and Olsson, {Richard F.} and Muthalagu Ramanathan and Saad, {Ayman A.} and Melhem Solh and Celalettin Ustun and David Valc{\'a}rcel and Erica Warlick and Wirk, {Baldeep M.} and Matt Kalaycio and Edwin Alyea and Uday Popat and Ronald Sobecks and Wael Saber",

note = "Funding Information: Financial disclosure: The CIBMTR is supported by Public Health Service Grant/Cooperative Agreement 5U24-CA076518 from the National Cancer Institute (NCI), the National Heart, Lung and Blood Institute (NHLBI), and the National Institute of Allergy and Infectious Diseases (NIAID); Grant/Cooperative Agreement 5U10HL069294 from the NHLBI and NCI; Contract HHSH250201200016C) with the Health Resources and Services Administration (HRSA/DHHS); Grants N00014-13-1-0039 and N00014-14-1-0028 from the Office of Naval Research; and grants from Alexion; *Amgen, Inc.; Anonymous donation to the Medical College of Wisconsin; Be the Match Foundation; *Bristol Myers Squibb Oncology; *Celgene Corporation; *Chimerix, Inc.; Fred Hutchinson Cancer Research Center; Gamida Cell Ltd.; Genentech, Inc.; Genzyme Corporation; *Gilead Sciences, Inc.; Health Research, Inc. Roswell Park Cancer Institute; HistoGenetics, Inc.; Incyte Corporation; *Jazz Pharmaceuticals, Inc.; Jeff Gordon Children's Foundation; The Leukemia & Lymphoma Society; The Medical College of Wisconsin; Merck & Co, Inc.; Mesoblast; *Millennium: The Takeda Oncology Co.; *Miltenyi Biotec, Inc.; National Marrow Donor Program; Neovii Biotech NA, Inc.; Novartis Pharmaceuticals Corporation; Onyx Pharmaceuticals; Optum Healthcare Solutions, Inc.; Otsuka America Pharmaceutical, Inc.; Otsuka Pharmaceutical Co, Ltd. – Japan; Oxford Immunotec; Perkin Elmer, Inc.; Pharmacyclics; *Sanofi US; Seattle Genetics; Sigma-Tau Pharmaceuticals; *Spectrum Pharmaceuticals, Inc.; St. Baldrick's Foundation; *Sunesis Pharmaceuticals, Inc.; Swedish Orphan Biovitrum, Inc.; Telomere Diagnostics, Inc.; TerumoBCT; Therakos, Inc.; University of Minnesota; and *Wellpoint, Inc. The views expressed in this article do not reflect the official policy or position of the National Institutes of Health, the Department of the Navy, the Department of Defense, the Health Resources and Services Administration, or any other agency of the US Government. Publisher Copyright: {\textcopyright} 2017 The American Society for Blood and Marrow Transplantation",

year = "2017",

month = jun,

doi = "10.1016/j.bbmt.2017.03.014",

language = "English (US)",

volume = "23",

pages = "971--979",

journal = "Biology of Blood and Marrow Transplantation",

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TY - JOUR

T1 - Outcomes after Umbilical Cord Blood Transplantation for Myelodysplastic Syndromes

AU - Gerds, Aaron T.

AU - Woo Ahn, Kwang

AU - Hu, Zhen Huan

AU - Abdel-Azim, Hisham

AU - Akpek, Gorgun

AU - Aljurf, Mahmoud

AU - Ballen, Karen K.

AU - Beitinjaneh, Amer

AU - Bacher, Ulrike

AU - Cahn, Jean Yves

AU - Chhabra, Saurabh

AU - Cutler, Corey

AU - Daly, Andrew

AU - DeFilipp, Zachariah

AU - Gale, Robert Peter

AU - Gergis, Usama

AU - Grunwald, Michael R.

AU - Hale, Gregory A.

AU - Hamilton, Betty Ky

AU - Jagasia, Madan

AU - Kamble, Rammurti T.

AU - Kindwall-Keller, Tamila

AU - Nishihori, Taiga

AU - Olsson, Richard F.

AU - Ramanathan, Muthalagu

AU - Saad, Ayman A.

AU - Solh, Melhem

AU - Ustun, Celalettin

AU - Valcárcel, David

AU - Warlick, Erica

AU - Wirk, Baldeep M.

AU - Kalaycio, Matt

AU - Alyea, Edwin

AU - Popat, Uday

AU - Sobecks, Ronald

AU - Saber, Wael

N1 - Funding Information: Financial disclosure: The CIBMTR is supported by Public Health Service Grant/Cooperative Agreement 5U24-CA076518 from the National Cancer Institute (NCI), the National Heart, Lung and Blood Institute (NHLBI), and the National Institute of Allergy and Infectious Diseases (NIAID); Grant/Cooperative Agreement 5U10HL069294 from the NHLBI and NCI; Contract HHSH250201200016C) with the Health Resources and Services Administration (HRSA/DHHS); Grants N00014-13-1-0039 and N00014-14-1-0028 from the Office of Naval Research; and grants from Alexion; *Amgen, Inc.; Anonymous donation to the Medical College of Wisconsin; Be the Match Foundation; *Bristol Myers Squibb Oncology; *Celgene Corporation; *Chimerix, Inc.; Fred Hutchinson Cancer Research Center; Gamida Cell Ltd.; Genentech, Inc.; Genzyme Corporation; *Gilead Sciences, Inc.; Health Research, Inc. Roswell Park Cancer Institute; HistoGenetics, Inc.; Incyte Corporation; *Jazz Pharmaceuticals, Inc.; Jeff Gordon Children's Foundation; The Leukemia & Lymphoma Society; The Medical College of Wisconsin; Merck & Co, Inc.; Mesoblast; *Millennium: The Takeda Oncology Co.; *Miltenyi Biotec, Inc.; National Marrow Donor Program; Neovii Biotech NA, Inc.; Novartis Pharmaceuticals Corporation; Onyx Pharmaceuticals; Optum Healthcare Solutions, Inc.; Otsuka America Pharmaceutical, Inc.; Otsuka Pharmaceutical Co, Ltd. – Japan; Oxford Immunotec; Perkin Elmer, Inc.; Pharmacyclics; *Sanofi US; Seattle Genetics; Sigma-Tau Pharmaceuticals; *Spectrum Pharmaceuticals, Inc.; St. Baldrick's Foundation; *Sunesis Pharmaceuticals, Inc.; Swedish Orphan Biovitrum, Inc.; Telomere Diagnostics, Inc.; TerumoBCT; Therakos, Inc.; University of Minnesota; and *Wellpoint, Inc. The views expressed in this article do not reflect the official policy or position of the National Institutes of Health, the Department of the Navy, the Department of Defense, the Health Resources and Services Administration, or any other agency of the US Government. Publisher Copyright: © 2017 The American Society for Blood and Marrow Transplantation

PY - 2017/6

Y1 - 2017/6

N2 - For patients with hematologic malignancies undergoing allogeneic hematopoietic cell transplantation, umbilical cord blood transplantation (UCBT) has become an acceptable alternative donor source in the absence of a matched sibling or unrelated donor. To date, however, there have been few published series dedicated solely to describing the outcomes of adult patients with myelodysplastic syndrome (MDS) who have undergone UCBT. Between 2004 and 2013, 176 adults with MDS underwent UCBT as reported to the Center for International Blood and Marrow Transplant Research. Median age at the time of transplantation was 56 years (range, 18-73 years). The study group included 10% with very low, 23% with low, 19% with intermediate, 19% with high, and 13% with very high-risk Revised International Prognostic Scoring System (IPSS-R) scores. The 100-day probability of grade II-IV acute graft-versus-host disease (GVHD) was 38%, and the 3-year probability of chronic GVHD was 28%. The probabilities of relapse and transplantation-related mortality (TRM) at 3 years were 32% and 40%, respectively, leading to a 3-year disease-free survival (DFS) of 28% and an overall survival (OS) of 31%. In multivariate analysis, increasing IPSS-R score at the time of HCT was associated with inferior TRM (P = .0056), DFS (P = .018), and OS (P = .0082), but not with GVHD or relapse. The presence of pretransplantation comorbidities was associated with TRM (P = .001), DFS (P = .02), and OS (P = .001). Reduced-intensity conditioning was associated with increased risk of relapse (relative risk, 3.95; 95% confidence interval, 1.78-8.75; P < .001), and although a higher proportion of myeloablative UCBTs were performed in patients with high-risk disease, the effect of conditioning regimen intensity was the same regardless of IPSS-R score. For some individuals who lack a matched sibling or unrelated donor, UCBT can result in long-term DFS; however, the success of UCBT in this population is hampered by a high rate of TRM.

AB - For patients with hematologic malignancies undergoing allogeneic hematopoietic cell transplantation, umbilical cord blood transplantation (UCBT) has become an acceptable alternative donor source in the absence of a matched sibling or unrelated donor. To date, however, there have been few published series dedicated solely to describing the outcomes of adult patients with myelodysplastic syndrome (MDS) who have undergone UCBT. Between 2004 and 2013, 176 adults with MDS underwent UCBT as reported to the Center for International Blood and Marrow Transplant Research. Median age at the time of transplantation was 56 years (range, 18-73 years). The study group included 10% with very low, 23% with low, 19% with intermediate, 19% with high, and 13% with very high-risk Revised International Prognostic Scoring System (IPSS-R) scores. The 100-day probability of grade II-IV acute graft-versus-host disease (GVHD) was 38%, and the 3-year probability of chronic GVHD was 28%. The probabilities of relapse and transplantation-related mortality (TRM) at 3 years were 32% and 40%, respectively, leading to a 3-year disease-free survival (DFS) of 28% and an overall survival (OS) of 31%. In multivariate analysis, increasing IPSS-R score at the time of HCT was associated with inferior TRM (P = .0056), DFS (P = .018), and OS (P = .0082), but not with GVHD or relapse. The presence of pretransplantation comorbidities was associated with TRM (P = .001), DFS (P = .02), and OS (P = .001). Reduced-intensity conditioning was associated with increased risk of relapse (relative risk, 3.95; 95% confidence interval, 1.78-8.75; P < .001), and although a higher proportion of myeloablative UCBTs were performed in patients with high-risk disease, the effect of conditioning regimen intensity was the same regardless of IPSS-R score. For some individuals who lack a matched sibling or unrelated donor, UCBT can result in long-term DFS; however, the success of UCBT in this population is hampered by a high rate of TRM.

KW - Blood and marrow transplantation

KW - Myelodysplastic syndrome

KW - Umbilical cord transplantation

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Outcomes after Umbilical Cord Blood Transplantation for Myelodysplastic Syndromes

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