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<div class="pre-content"><div><div class="bk_prnt"><p class="small">NCBI Bookshelf. A service of the National Library of Medicine, National Institutes of Health.</p><p>PDQ Cancer Information Summaries [Internet]. Bethesda (MD): National Cancer Institute (US); 2002-. </p></div><div class="iconblock clearfix whole_rhythm no_top_margin bk_noprnt"><a class="img_link icnblk_img" title="Table of Contents Page" href="/books/n/pdqcis/"><img class="source-thumb" src="/corehtml/pmc/pmcgifs/bookshelf/thumbs/th-pdqcis-lrg.png" alt="Cover of PDQ Cancer Information Summaries" height="100px" width="80px" /></a><div class="icnblk_cntnt eight_col"><h2>PDQ Cancer Information Summaries [Internet].</h2><a data-jig="ncbitoggler" href="#__NBK577657_dtls__">Show details</a><div style="display:none" class="ui-widget" id="__NBK577657_dtls__"><div>Bethesda (MD): <a href="http://www.cancer.gov/" ref="pagearea=page-banner&targetsite=external&targetcat=link&targettype=publisher">National Cancer Institute (US)</a>; 2002-.</div></div><div class="half_rhythm"></div><div class="bk_noprnt"><form method="get" action="/books/n/pdqcis/" id="bk_srch"><div class="bk_search"><label for="bk_term" class="offscreen_noflow">Search term</label><input type="text" title="Search this book" id="bk_term" name="term" value="" data-jig="ncbiclearbutton" /> <input type="submit" class="jig-ncbibutton" value="Search this book" submit="false" style="padding: 0.1em 0.4em;" /></div></form></div></div></div></div></div>
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<div class="main-content lit-style" itemscope="itemscope" itemtype="http://schema.org/CreativeWork"><div class="meta-content fm-sec"><h1 id="_NBK577657_"><span class="title" itemprop="name">Pediatric Autologous Hematopoietic Stem Cell Transplant (PDQ®)</span></h1><div class="subtitle whole_rhythm">Health Professional Version</div><p class="contrib-group"><span itemprop="author">PDQ Pediatric Treatment Editorial Board</span>.</p><p class="small">Published online: December 20, 2023.</p><p class="small">Created: <span itemprop="datePublished">February 4, 2022</span>.</p></div><div class="jig-ncbiinpagenav body-content whole_rhythm" data-jigconfig="allHeadingLevels: ['h2'],smoothScroll: false" itemprop="text"><div id="_abs_rndgid_" itemprop="description"><p id="CDR0000805686__441">This PDQ cancer information summary for health professionals provides comprehensive, peer-reviewed, evidence-based information about the use of autologous hematopoietic stem cell transplant in treating pediatric cancer. It is intended as a resource to inform and assist clinicians in the care of their patients. It does not provide formal guidelines or recommendations for making health care decisions.</p><p id="CDR0000805686__442">This summary is reviewed regularly and updated as necessary by the PDQ Pediatric Treatment Editorial Board, which is editorially independent of the National Cancer Institute (NCI). The summary reflects an independent review of the literature and does not represent a policy statement of NCI or the National Institutes of Health (NIH).</p></div><div id="CDR0000805686__644"><h2 id="_CDR0000805686__644_">Collection and Storage of Autologous Hematopoietic Stem Cells</h2><p id="CDR0000805686__645">Autologous hematopoietic stem cell transplant (HSCT) procedures require collection of growth-factor–mobilized peripheral blood stem cells (PBSCs) from patients using leukapheresis. Bone marrow can be used for autologous transplants, but PBSCs lead to quicker blood count recovery, resulting in less transplant-related toxicity. </p><p id="CDR0000805686__646">Patients being considered for autologous HSCT are generally given chemotherapy to determine tumor responsiveness and minimize the risk of tumor contamination in their bone marrow. After a number of rounds of chemotherapy, patients undergo the leukapheresis procedure, either as their blood counts recover from chemotherapy or during a break between chemotherapy treatments. Growth factors such as granulocyte colony-stimulating factor are used to increase the number of circulating stem and progenitor cells (CD34+ cells). Collection centers monitor the CD34-positive number in the patient and product each day to determine the best time to begin collection and when collection is complete. Patients with low numbers of CD34-positive cells before collection can often have their cells successfully collected using alternative mobilization approaches (e.g., addition of plerixafor).[<a class="bk_pop" href="#CDR0000805686_rl_644_1">1</a>] The collected PBSCs are cryopreserved for later use. After completion of an intensive preparative regimen using high-dose chemotherapy, which varies according to the tumor type, the PBSCs are administered to the patient at the time of transplant.</p><div id="CDR0000805686_rl_644"><h3>References</h3><ol><li><div class="bk_ref" id="CDR0000805686_rl_644_1">Patel B, Pearson H, Zacharoulis S: Mobilisation of haematopoietic stem cells in paediatric patients, prior to autologous transplantation following administration of plerixafor and G-CSF. Pediatr Blood Cancer 62 (8): 1477-80, 2015. [<a href="https://pubmed.ncbi.nlm.nih.gov/25755177" ref="pagearea=cite-ref&targetsite=entrez&targetcat=link&targettype=pubmed">PubMed<span class="bk_prnt">: 25755177</span></a>]</div></li></ol></div></div><div id="CDR0000805686__647"><h2 id="_CDR0000805686__647_">General Indications and Considerations for Autologous Procedures</h2><div id="CDR0000805686__654"><h3>Autologous Hematopoietic Stem Cell Transplant (HSCT) Indications for Solid Tumors and Lymphomas</h3><p id="CDR0000805686__656">In pediatrics, the most common autologous HSCT indications are for the treatment of some solid tumors and lymphomas.</p><p id="CDR0000805686__608">Autologous transplants have also been used to reset the immune system in patients with severe autoimmune disorders and to enable engraftment of genetically modified autologous hematopoietic stem cell progenitors to correct or ameliorate inherited disorders (e.g., immunodeficiencies, metabolic disorders, and hemoglobinopathies). These indications are not covered in this summary.</p><p id="CDR0000805686__655">Indications for HSCT vary over time as risk classifications for a given malignancy change and the efficacy of primary therapy improves. It is best to include specific indications in the context of complete therapy for any given disease.</p><p id="CDR0000805686__657"> With this in mind, links to sections in specific summaries that cover the most common pediatric autologous HSCT indications are provided below.</p><ol id="CDR0000805686__642"><li class="half_rhythm"><div>
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<b>Neuroblastoma.</b>
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<ul id="CDR0000805686__658"><li class="half_rhythm"><div>For more information, see the sections on <a href="/books/n/pdqcis/CDR0000062786/#CDR0000062786__214">Treatment of High-Risk Neuroblastoma</a> and <a href="/books/n/pdqcis/CDR0000062786/#CDR0000062786__138">Recurrent Neuroblastoma in Patients Initially Classified as High Risk</a> in Neuroblastoma Treatment.</div></li></ul>
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</div></li><li class="half_rhythm"><div><b>Brain tumors.</b> Indications for young patients to reduce or eliminate cranial radiation therapy; indications for responsive tumors at relapse.<ul id="CDR0000805686__659"><li class="half_rhythm"><div>For more information, see the <a href="/books/n/pdqcis/CDR0000614165/#CDR0000614165__322">Treatment of Pediatric-Type Diffuse High-Grade Gliomas</a> section in Childhood Astrocytomas, Other Gliomas, and Glioneuronal/Neuronal Tumors Treatment.</div></li><li class="half_rhythm"><div>For more information, see the <a href="/books/n/pdqcis/CDR0000587224/#CDR0000587224__29">Treatment of Childhood CNS Atypical Teratoid/Rhabdoid Tumor</a> section in Childhood Central Nervous System Atypical Teratoid/Rhabdoid Tumor Treatment.</div></li><li class="half_rhythm"><div>For more information, see the sections on <a href="/books/n/pdqcis/CDR0000548358/#CDR0000548358__1914">Treatment of Childhood Medulloblastoma</a>, <a href="/books/n/pdqcis/CDR0000548358/#CDR0000548358__1968">Treatment of Childhood Pineoblastoma</a>, and <a href="/books/n/pdqcis/CDR0000548358/#CDR0000548358__71">Treatment of Recurrent Childhood Medulloblastoma and Other CNS Embryonal Tumors</a> in Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors Treatment.</div></li></ul></div></li><li class="half_rhythm"><div>
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<b>Germ cell tumors (GCTs) (intracranial and extracranial).</b>
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<ul id="CDR0000805686__660"><li class="half_rhythm"><div>For more information, see the <a href="/books/n/pdqcis/CDR0000062854/#CDR0000062854__715">Nonstandard Treatment Options for Recurrent Malignant GCTs in Children</a> section in Childhood Extracranial Germ Cell Tumors Treatment.</div></li><li class="half_rhythm"><div>For more information, see the <a href="/books/n/pdqcis/CDR0000712041/#CDR0000712041__47">Treatment of Recurrent Childhood CNS Germ Cell Tumors</a> section in Childhood Central Nervous System Germ Cell Tumors Treatment.</div></li></ul>
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</div></li><li class="half_rhythm"><div>
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<b>Retinoblastoma.</b>
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<ul id="CDR0000805686__661"><li class="half_rhythm"><div>For more information, see the sections on <a href="/books/n/pdqcis/CDR0000062846/#CDR0000062846__541">Treatment of CNS Disease</a>, <a href="/books/n/pdqcis/CDR0000062846/#CDR0000062846__543">Treatment of Synchronous Trilateral Retinoblastoma</a>, <a href="/books/n/pdqcis/CDR0000062846/#CDR0000062846__546">Treatment of Extracranial Metastatic Retinoblastoma</a>, and <a href="/books/n/pdqcis/CDR0000062846/#CDR0000062846__849">Treatment of Progressive or Recurrent Extraocular Retinoblastoma</a> in Retinoblastoma Treatment.</div></li></ul>
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</div></li><li class="half_rhythm"><div>
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<b>Hodgkin lymphoma.</b>
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<ul id="CDR0000805686__662"><li class="half_rhythm"><div>For more information, see the <a href="/books/n/pdqcis/CDR0000062933/#CDR0000062933__113">Treatment of Primary Refractory or Recurrent Hodgkin Lymphoma in Children and Adolescents</a> section in Childhood Hodgkin Lymphoma Treatment.</div></li></ul>
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</div></li><li class="half_rhythm"><div>
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<b>Non-Hodgkin lymphoma.</b>
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<ul id="CDR0000805686__663"><li class="half_rhythm"><div>For more information, see the sections on <a href="/books/n/pdqcis/CDR0000062808/#CDR0000062808__797">Treatment options for recurrent or refractory Burkitt lymphoma/leukemia</a>, <a href="/books/n/pdqcis/CDR0000062808/#CDR0000062808__827">Treatment Options for Recurrent or Refractory Lymphoblastic Lymphoma</a>, <a href="/books/n/pdqcis/CDR0000062808/#CDR0000062808__839">Treatment Options for Recurrent or Refractory Anaplastic Large Cell Lymphoma</a>, <a href="/books/n/pdqcis/CDR0000062808/#CDR0000062808__1652">Treatment options for lymphoproliferative disease associated with primary immunodeficiency</a>, <a href="/books/n/pdqcis/CDR0000062808/#CDR0000062808__869">Treatment options for peripheral T-cell lymphoma</a>, and <a href="/books/n/pdqcis/CDR0000062808/#CDR0000062808__875">Treatment options for cutaneous T-cell lymphoma</a> in Childhood Non-Hodgkin Lymphoma Treatment.</div></li></ul>
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</div></li></ol><p id="CDR0000805686__653"> For autologous transplants to result in cure of malignancies, the following must apply:</p><ul id="CDR0000805686__156"><li class="half_rhythm"><div>A dose-intensified chemotherapy regimen (with or without radiation therapy) with hematopoietic stem cell support is used to achieve a significantly higher cell kill than could be achieved without the use of hematopoietic stem cell support. This approach may include increased tumor kill in areas where standard-dose chemotherapy has less penetration (central nervous system).</div></li><li class="half_rhythm"><div>Meaningful percentages of cure or long-term remission from the disease must occur without significant nonhematopoietic toxicities that would otherwise limit the therapeutic benefit achieved.</div></li></ul><p id="CDR0000805686__651">The tumor-specific activity and intensity of agents used for autologous regimens have been shown to be important in improving survival. </p><p id="CDR0000805686__652">The contamination of the collected stem cell product by persistent tumor cells is one concern with autologous approaches for these and other tumor types. Although many techniques have been developed to remove or purge tumor cells from products, studies have shown no benefit to tumor purging.[<a class="bk_pop" href="#CDR0000805686_rl_647_1">1</a>]</p></div><div id="CDR0000805686_rl_647"><h3>References</h3><ol><li><div class="bk_ref" id="CDR0000805686_rl_647_1">Kreissman SG, Seeger RC, Matthay KK, et al.: Purged versus non-purged peripheral blood stem-cell transplantation for high-risk neuroblastoma (COG A3973): a randomised phase 3 trial. Lancet Oncol 14 (10): 999-1008, 2013. [<a href="/pmc/articles/PMC3963485/" ref="pagearea=cite-ref&targetsite=entrez&targetcat=link&targettype=pmc">PMC free article<span class="bk_prnt">: PMC3963485</span></a>] [<a href="https://pubmed.ncbi.nlm.nih.gov/23890779" ref="pagearea=cite-ref&targetsite=entrez&targetcat=link&targettype=pubmed">PubMed<span class="bk_prnt">: 23890779</span></a>]</div></li></ol></div></div><div id="CDR0000805686__5"><h2 id="_CDR0000805686__5_">Latest Updates to This Summary (12/20/2023)</h2><p id="CDR0000805686__96">The PDQ cancer information summaries are reviewed regularly and updated as new information becomes available. This section describes the latest changes made to this summary as of the date above.</p><p id="CDR0000805686__664">Editorial changes were made to this summary.</p><p id="CDR0000805686__disclaimerHP_3">This summary is written and maintained by the <a href="https://www.cancer.gov/publications/pdq/editorial-boards/pediatric-treatment" ref="pagearea=body&targetsite=external&targetcat=link&targettype=uri">PDQ Pediatric Treatment Editorial Board</a>, which is
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editorially independent of NCI. The summary reflects an independent review of
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the literature and does not represent a policy statement of NCI or NIH. More
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information about summary policies and the role of the PDQ Editorial Boards in
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maintaining the PDQ summaries can be found on the <a href="#CDR0000805686__AboutThis_1">About This PDQ Summary</a> and <a href="https://www.cancer.gov/publications/pdq" ref="pagearea=body&targetsite=external&targetcat=link&targettype=uri">PDQ® Cancer Information for Health Professionals</a> pages.
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</p></div><div id="CDR0000805686__AboutThis_1"><h2 id="_CDR0000805686__AboutThis_1_">About This PDQ Summary</h2><div id="CDR0000805686__AboutThis_2"><h3>Purpose of This Summary</h3><p id="CDR0000805686__AboutThis_3">This PDQ cancer information summary for health professionals provides comprehensive, peer-reviewed, evidence-based information about the use of autologous hematopoietic stem cell transplant in treating pediatric cancer. It is intended as a resource to inform and assist clinicians in the care of their patients. It does not provide formal guidelines or recommendations for making health care decisions.</p></div><div id="CDR0000805686__AboutThis_4"><h3>Reviewers and Updates</h3><p id="CDR0000805686__AboutThis_5">This summary is reviewed regularly and updated as necessary by the <a href="https://www.cancer.gov/publications/pdq/editorial-boards/pediatric-treatment" ref="pagearea=body&targetsite=external&targetcat=link&targettype=uri">PDQ Pediatric Treatment Editorial Board</a>, which is editorially independent of the National Cancer Institute (NCI). The summary reflects an independent review of the literature and does not represent a policy statement of NCI or the National Institutes of Health (NIH).</p><p id="CDR0000805686__AboutThis_22"> Board members review recently published articles each month to determine whether an article should:</p><ul id="CDR0000805686__AboutThis_6"><li class="half_rhythm"><div>be discussed at a meeting,</div></li><li class="half_rhythm"><div>be cited with text, or</div></li><li class="half_rhythm"><div>replace or update an existing article that is already cited.</div></li></ul><p id="CDR0000805686__AboutThis_7">Changes to the summaries are made through a consensus process in which Board members evaluate the strength of the evidence in the published articles and determine how the article should be included in the summary.</p><p>The lead reviewers for Pediatric Autologous Hematopoietic Stem Cell Transplant are:</p><ul><li class="half_rhythm"><div>Thomas G. Gross, MD, PhD (National Cancer Institute)</div></li><li class="half_rhythm"><div>Michael A. Pulsipher, MD (Children's Hospital Los Angeles)</div></li></ul><p id="CDR0000805686__AboutThis_9">Any comments or questions about the summary content should be submitted to Cancer.gov through the NCI website's <a href="https://www.cancer.gov/contact/email-us" ref="pagearea=body&targetsite=external&targetcat=link&targettype=uri">Email Us</a>. Do not contact the individual Board Members with questions or comments about the summaries. Board members will not respond to individual inquiries.</p></div><div id="CDR0000805686__AboutThis_10"><h3>Levels of Evidence</h3><p id="CDR0000805686__AboutThis_11">Some of the reference citations in this summary are accompanied by a level-of-evidence designation. These designations are intended to help readers assess the strength of the evidence supporting the use of specific interventions or approaches. The PDQ Pediatric Treatment Editorial Board uses a <a href="/books/n/pdqcis/CDR0000062796/">formal evidence ranking system</a> in developing its level-of-evidence designations.</p></div><div id="CDR0000805686__AboutThis_12"><h3>Permission to Use This Summary</h3><p id="CDR0000805686__AboutThis_13">PDQ is a registered trademark. Although the content of PDQ documents can be used freely as text, it cannot be identified as an NCI PDQ cancer information summary unless it is presented in its entirety and is regularly updated. However, an author would be permitted to write a sentence such as “NCI’s PDQ cancer information summary about breast cancer prevention states the risks succinctly: [include excerpt from the summary].”</p><p id="CDR0000805686__AboutThis_14">The preferred citation for this PDQ summary is:</p><p id="CDR0000805686__AboutThis_15">PDQ® Pediatric Treatment Editorial Board. PDQ Pediatric Autologous Hematopoietic Stem Cell Transplant. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: <a href="https://www.cancer.gov/types/childhood-cancers/hp-stem-cell-transplant/autologous" ref="pagearea=body&targetsite=external&targetcat=link&targettype=uri">https://www.cancer.gov/types/childhood-cancers/hp-stem-cell-transplant/autologous</a>. Accessed <MM/DD/YYYY>. [PMID: 35133767]</p><p id="CDR0000805686__AboutThis_16">Images in this summary are used with permission of the author(s), artist, and/or publisher for use within the PDQ summaries only. Permission to use images outside the context of PDQ information must be obtained from the owner(s) and cannot be granted by the National Cancer Institute. Information about using the illustrations in this summary, along with many other cancer-related images, is available in <a href="https://visualsonline.cancer.gov/" ref="pagearea=body&targetsite=external&targetcat=link&targettype=uri">Visuals Online</a>, a collection of over 2,000 scientific images.
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</p></div><div id="CDR0000805686__AboutThis_17"><h3>Disclaimer</h3><p id="CDR0000805686__AboutThis_18">Based on the strength of the available evidence, treatment options may be described as either “standard” or “under clinical evaluation.” These classifications should not be used as a basis for insurance reimbursement determinations. More information on insurance coverage is available on Cancer.gov on the <a href="https://www.cancer.gov/about-cancer/managing-care" ref="pagearea=body&targetsite=external&targetcat=link&targettype=uri">Managing Cancer Care</a> page.</p></div><div id="CDR0000805686__AboutThis_20"><h3>Contact Us</h3><p id="CDR0000805686__AboutThis_21">More information about contacting us or receiving help with the Cancer.gov website can be found on our <a href="https://www.cancer.gov/contact" ref="pagearea=body&targetsite=external&targetcat=link&targettype=uri">Contact Us for Help</a> page. Questions can also be submitted to Cancer.gov through the website’s <a href="https://www.cancer.gov/contact/email-us" ref="pagearea=body&targetsite=external&targetcat=link&targettype=uri">Email Us</a>.</p></div></div></div></div>
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<div xmlns:np="http://ncbi.gov/portal/XSLT/namespace" xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance"></div><div class="portlet"><div class="portlet_head"><div class="portlet_title"><h3><span>Views</span></h3></div><a name="Shutter" sid="1" href="#" class="portlet_shutter" title="Show/hide content" remembercollapsed="true" pgsec_name="PDF_download" id="Shutter"></a></div><div class="portlet_content"><ul xmlns:np="http://ncbi.gov/portal/XSLT/namespace" xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" class="simple-list"><li><a href="/books/NBK577657.3/?report=reader">PubReader</a></li><li><a href="/books/NBK577657.3/?report=printable">Print View</a></li><li><a data-jig="ncbidialog" href="#_ncbi_dlg_citbx_NBK577657" data-jigconfig="width:400,modal:true">Cite this Page</a><div id="_ncbi_dlg_citbx_NBK577657" style="display:none" title="Cite this Page"><div class="bk_tt">PDQ Pediatric Treatment Editorial Board. Pediatric Autologous Hematopoietic Stem Cell Transplant (PDQ®): Health Professional Version. 2023 Dec 20. In: PDQ Cancer Information Summaries [Internet]. Bethesda (MD): National Cancer Institute (US); 2002-. <span class="bk_cite_avail"></span></div></div></li></ul></div></div><div class="portlet"><div class="portlet_head"><div class="portlet_title"><h3><span>Version History</span></h3></div><a name="Shutter" sid="1" href="#" class="portlet_shutter shutter_closed" title="Show/hide content" remembercollapsed="true" pgsec_name="version_history" id="Shutter"></a></div><div class="portlet_content" style="display: none;"><ul xmlns:np="http://ncbi.gov/portal/XSLT/namespace" xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" class="simple-list"><li><span class="bk_col_itm"><a href="/books/NBK577657.4/">NBK577657.4</a></span> June 13, 2024</li><li><span class="bk_col_itm">NBK577657.3</span> December 20, 2023 (Displayed Version)</li><li><span class="bk_col_itm"><a href="/books/NBK577657.2/">NBK577657.2</a></span> December 12, 2023</li><li><span class="bk_col_itm"><a href="/books/NBK577657.1/">NBK577657.1</a></span> February 4, 2022</li></ul></div></div><div class="portlet"><div class="portlet_head"><div class="portlet_title"><h3><span>In this Page</span></h3></div><a name="Shutter" sid="1" href="#" class="portlet_shutter" title="Show/hide content" remembercollapsed="true" pgsec_name="page-toc" id="Shutter"></a></div><div class="portlet_content"><ul xmlns:np="http://ncbi.gov/portal/XSLT/namespace" xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" class="simple-list"><li><a href="#CDR0000805686__644" ref="log$=inpage&link_id=inpage">Collection and Storage of Autologous Hematopoietic Stem Cells</a></li><li><a href="#CDR0000805686__647" ref="log$=inpage&link_id=inpage">General Indications and Considerations for Autologous Procedures</a></li><li><a href="#CDR0000805686__5" ref="log$=inpage&link_id=inpage">Latest Updates to This Summary (12/20/2023)</a></li><li><a href="#CDR0000805686__AboutThis_1" ref="log$=inpage&link_id=inpage">About This PDQ Summary</a></li></ul></div></div><div class="portlet"><div 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