Cargando…

Haploidentical Peripheral Blood Stem Cell Transplantation Demonstrates Stable Engraftment in Adults with Sickle Cell Disease

We report on the screening and development of haploidentical hematopoietic stem cell transplantation (HSCT) for adult patients with clinically aggressive sickle cell disease (SCD) at our institution. Of 50 adult SCD patients referred for HSCT between January 2014 and March 2017, 20% were denied by i...

Descripción completa

Detalles Bibliográficos
Autores principales: Saraf, Santosh L., Oh, Annie L., Patel, Pritesh R., Sweiss, Karen, Koshy, Matthew, Campbell-Lee, Sally, Gowhari, Michel, Jain, Shivi, Peace, David, Quigley, John G., Khan, Irum, Molokie, Robert E., Mahmud, Nadim, Gordeuk, Victor R., Rondelli, Damiano
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Blood and Marrow Transplantation. 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6108914/
https://www.ncbi.nlm.nih.gov/pubmed/29656137
http://dx.doi.org/10.1016/j.bbmt.2018.03.031
_version_ 1783350234076676096
author Saraf, Santosh L.
Oh, Annie L.
Patel, Pritesh R.
Sweiss, Karen
Koshy, Matthew
Campbell-Lee, Sally
Gowhari, Michel
Jain, Shivi
Peace, David
Quigley, John G.
Khan, Irum
Molokie, Robert E.
Mahmud, Nadim
Gordeuk, Victor R.
Rondelli, Damiano
author_facet Saraf, Santosh L.
Oh, Annie L.
Patel, Pritesh R.
Sweiss, Karen
Koshy, Matthew
Campbell-Lee, Sally
Gowhari, Michel
Jain, Shivi
Peace, David
Quigley, John G.
Khan, Irum
Molokie, Robert E.
Mahmud, Nadim
Gordeuk, Victor R.
Rondelli, Damiano
author_sort Saraf, Santosh L.
collection PubMed
description We report on the screening and development of haploidentical hematopoietic stem cell transplantation (HSCT) for adult patients with clinically aggressive sickle cell disease (SCD) at our institution. Of 50 adult SCD patients referred for HSCT between January 2014 and March 2017, 20% were denied by insurance. Of 41 patients initially screened, 10% lacked an available haploidentical donor, 29% had elevated donor-specific antibodies (DSAs), and 34% declined to proceed to HSCT. All 10 patients who were transplanted received peripheral blood stem cells. The initial 2 were conditioned with alemtuzumab/total body irradiation (TBI) 3 Gy followed by post-transplant cyclophosphamide and failed to engraft. The next 8 patients received the regimen developed at Johns Hopkins University with TBI 3 Gy. Granulocyte colony-stimulating factor was administered from day +12 in those with HbS < 30%. All 8 patients engrafted with a median time to neutrophil >.5 × 10(9)/L of 22 days (range, 18 to 23). One patient subsequently lost the graft, and 7 (87.5%) maintained >95% donor cell chimerism at 1-year post-HSCT. Two patients developed acute graft-versus-host disease (GVHD) of at least grade II. One had chronic GVHD and died >1 year after HSCT of unknown causes. With a median follow-up of 16 months (range, 11 to 29), 7 patients (87.5%) are alive. Our findings suggest that limited insurance coverage, high rate of DSAs, and patient declining HSCT may limit the availability of haploidentical HSCT in adult SCD patients. The modified Hopkins regimen used here demonstrates high engraftment and low morbidity rates and should be tested in larger, multicenter, prospective clinical trials.
format Online
Article
Text
id pubmed-6108914
institution National Center for Biotechnology Information
language English
publishDate 2018
publisher American Society for Blood and Marrow Transplantation.
record_format MEDLINE/PubMed
spelling pubmed-61089142019-08-01 Haploidentical Peripheral Blood Stem Cell Transplantation Demonstrates Stable Engraftment in Adults with Sickle Cell Disease Saraf, Santosh L. Oh, Annie L. Patel, Pritesh R. Sweiss, Karen Koshy, Matthew Campbell-Lee, Sally Gowhari, Michel Jain, Shivi Peace, David Quigley, John G. Khan, Irum Molokie, Robert E. Mahmud, Nadim Gordeuk, Victor R. Rondelli, Damiano Biol Blood Marrow Transplant Article We report on the screening and development of haploidentical hematopoietic stem cell transplantation (HSCT) for adult patients with clinically aggressive sickle cell disease (SCD) at our institution. Of 50 adult SCD patients referred for HSCT between January 2014 and March 2017, 20% were denied by insurance. Of 41 patients initially screened, 10% lacked an available haploidentical donor, 29% had elevated donor-specific antibodies (DSAs), and 34% declined to proceed to HSCT. All 10 patients who were transplanted received peripheral blood stem cells. The initial 2 were conditioned with alemtuzumab/total body irradiation (TBI) 3 Gy followed by post-transplant cyclophosphamide and failed to engraft. The next 8 patients received the regimen developed at Johns Hopkins University with TBI 3 Gy. Granulocyte colony-stimulating factor was administered from day +12 in those with HbS < 30%. All 8 patients engrafted with a median time to neutrophil >.5 × 10(9)/L of 22 days (range, 18 to 23). One patient subsequently lost the graft, and 7 (87.5%) maintained >95% donor cell chimerism at 1-year post-HSCT. Two patients developed acute graft-versus-host disease (GVHD) of at least grade II. One had chronic GVHD and died >1 year after HSCT of unknown causes. With a median follow-up of 16 months (range, 11 to 29), 7 patients (87.5%) are alive. Our findings suggest that limited insurance coverage, high rate of DSAs, and patient declining HSCT may limit the availability of haploidentical HSCT in adult SCD patients. The modified Hopkins regimen used here demonstrates high engraftment and low morbidity rates and should be tested in larger, multicenter, prospective clinical trials. American Society for Blood and Marrow Transplantation. 2018-08 2018-04-12 /pmc/articles/PMC6108914/ /pubmed/29656137 http://dx.doi.org/10.1016/j.bbmt.2018.03.031 Text en © 2018 American Society for Blood and Marrow Transplantation. Since January 2020 Elsevier has created a COVID-19 resource centre with free information in English and Mandarin on the novel coronavirus COVID-19. The COVID-19 resource centre is hosted on Elsevier Connect, the company's public news and information website. Elsevier hereby grants permission to make all its COVID-19-related research that is available on the COVID-19 resource centre - including this research content - immediately available in PubMed Central and other publicly funded repositories, such as the WHO COVID database with rights for unrestricted research re-use and analyses in any form or by any means with acknowledgement of the original source. These permissions are granted for free by Elsevier for as long as the COVID-19 resource centre remains active.
spellingShingle Article
Saraf, Santosh L.
Oh, Annie L.
Patel, Pritesh R.
Sweiss, Karen
Koshy, Matthew
Campbell-Lee, Sally
Gowhari, Michel
Jain, Shivi
Peace, David
Quigley, John G.
Khan, Irum
Molokie, Robert E.
Mahmud, Nadim
Gordeuk, Victor R.
Rondelli, Damiano
Haploidentical Peripheral Blood Stem Cell Transplantation Demonstrates Stable Engraftment in Adults with Sickle Cell Disease
title Haploidentical Peripheral Blood Stem Cell Transplantation Demonstrates Stable Engraftment in Adults with Sickle Cell Disease
title_full Haploidentical Peripheral Blood Stem Cell Transplantation Demonstrates Stable Engraftment in Adults with Sickle Cell Disease
title_fullStr Haploidentical Peripheral Blood Stem Cell Transplantation Demonstrates Stable Engraftment in Adults with Sickle Cell Disease
title_full_unstemmed Haploidentical Peripheral Blood Stem Cell Transplantation Demonstrates Stable Engraftment in Adults with Sickle Cell Disease
title_short Haploidentical Peripheral Blood Stem Cell Transplantation Demonstrates Stable Engraftment in Adults with Sickle Cell Disease
title_sort haploidentical peripheral blood stem cell transplantation demonstrates stable engraftment in adults with sickle cell disease
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6108914/
https://www.ncbi.nlm.nih.gov/pubmed/29656137
http://dx.doi.org/10.1016/j.bbmt.2018.03.031
work_keys_str_mv AT sarafsantoshl haploidenticalperipheralbloodstemcelltransplantationdemonstratesstableengraftmentinadultswithsicklecelldisease
AT ohanniel haploidenticalperipheralbloodstemcelltransplantationdemonstratesstableengraftmentinadultswithsicklecelldisease
AT patelpriteshr haploidenticalperipheralbloodstemcelltransplantationdemonstratesstableengraftmentinadultswithsicklecelldisease
AT sweisskaren haploidenticalperipheralbloodstemcelltransplantationdemonstratesstableengraftmentinadultswithsicklecelldisease
AT koshymatthew haploidenticalperipheralbloodstemcelltransplantationdemonstratesstableengraftmentinadultswithsicklecelldisease
AT campbellleesally haploidenticalperipheralbloodstemcelltransplantationdemonstratesstableengraftmentinadultswithsicklecelldisease
AT gowharimichel haploidenticalperipheralbloodstemcelltransplantationdemonstratesstableengraftmentinadultswithsicklecelldisease
AT jainshivi haploidenticalperipheralbloodstemcelltransplantationdemonstratesstableengraftmentinadultswithsicklecelldisease
AT peacedavid haploidenticalperipheralbloodstemcelltransplantationdemonstratesstableengraftmentinadultswithsicklecelldisease
AT quigleyjohng haploidenticalperipheralbloodstemcelltransplantationdemonstratesstableengraftmentinadultswithsicklecelldisease
AT khanirum haploidenticalperipheralbloodstemcelltransplantationdemonstratesstableengraftmentinadultswithsicklecelldisease
AT molokieroberte haploidenticalperipheralbloodstemcelltransplantationdemonstratesstableengraftmentinadultswithsicklecelldisease
AT mahmudnadim haploidenticalperipheralbloodstemcelltransplantationdemonstratesstableengraftmentinadultswithsicklecelldisease
AT gordeukvictorr haploidenticalperipheralbloodstemcelltransplantationdemonstratesstableengraftmentinadultswithsicklecelldisease
AT rondellidamiano haploidenticalperipheralbloodstemcelltransplantationdemonstratesstableengraftmentinadultswithsicklecelldisease