Literature DB >> 31697818

How I treat sickle cell disease with hematopoietic cell transplantation.

Elizabeth O Stenger1, Shalini Shenoy2, Lakshmanan Krishnamurti3.   

Abstract

Sickle cell disease (SCD) leads to significant morbidity and early mortality, and hematopoietic cell transplantation (HCT) is the only widely available cure, with impacts seen on SCD-related organ dysfunction. Outcomes are excellent following matched-related donor (MRD) HCT, leading to significantly expanded application of this treatment over the past decade. The majority of SCD patients lack an MRD, but outcomes following alternative donor HCT continue to improve on clinical trials. Within this framework, we aim to provide our perspective on how to apply research findings to clinical practice, for an individual patient. We also emphasize that the preparation of SCD recipients for HCT and supporting them through HCT have special nuances that require awareness and close attention. Through the use of clinical vignettes, we provide our perpsective on the complex decision-making process in HCT for SCD as well as recommendations for the evaluation and support of these patients through HCT.
© 2019 by The American Society of Hematology.

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Year:  2019        PMID: 31697818      PMCID: PMC6923666          DOI: 10.1182/blood.2019000821

Source DB:  PubMed          Journal:  Blood        ISSN: 0006-4971            Impact factor:   22.113


  94 in total

Review 1.  When there is no match, the game is not over: Alternative donor options for hematopoietic stem cell transplantation in sickle cell disease.

Authors:  Jacinth J Joseph; Allistair A Abraham; Courtney D Fitzhugh
Journal:  Semin Hematol       Date:  2018-05-03       Impact factor: 3.851

2.  Treosulfan-based conditioning regimen for allogeneic haematopoietic stem cell transplantation in children with sickle cell disease.

Authors:  Luisa Strocchio; Marco Zecca; Patrizia Comoli; Tommaso Mina; Giovanna Giorgiani; Eugenia Giraldi; Luciana Vinti; Pietro Merli; Mario Regazzi; Franco Locatelli
Journal:  Br J Haematol       Date:  2015-03-27       Impact factor: 6.998

3.  Cyclophosphamide improves engraftment in patients with SCD and severe organ damage who undergo haploidentical PBSCT.

Authors:  Courtney D Fitzhugh; Matthew M Hsieh; Tiffani Taylor; Wynona Coles; Katherine Roskom; Delon Wilson; Elizabeth Wright; Neal Jeffries; Christopher J Gamper; Jonathan Powell; Leo Luznik; John F Tisdale
Journal:  Blood Adv       Date:  2017-04-19

4.  Functional and Radiologic Assessment of the Brain after Reduced-Intensity Unrelated Donor Transplantation for Severe Sickle Cell Disease: Blood and Marrow Transplant Clinical Trials Network Study 0601.

Authors:  Allison A King; Robert C McKinstry; Juan Wu; Mary Eapen; Regina Abel; Taniya Varughese; Naynesh Kamani; Shalini Shenoy
Journal:  Biol Blood Marrow Transplant       Date:  2019-01-11       Impact factor: 5.742

5.  Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel.

Authors:  Emanuele Angelucci; Susanne Matthes-Martin; Donatella Baronciani; Françoise Bernaudin; Sonia Bonanomi; Maria Domenica Cappellini; Jean-Hugues Dalle; Paolo Di Bartolomeo; Cristina Díaz de Heredia; Roswitha Dickerhoff; Claudio Giardini; Eliane Gluckman; Ayad Achmed Hussein; Naynesh Kamani; Milen Minkov; Franco Locatelli; Vanderson Rocha; Petr Sedlacek; Frans Smiers; Isabelle Thuret; Isaac Yaniv; Marina Cavazzana; Christina Peters
Journal:  Haematologica       Date:  2014-05       Impact factor: 9.941

6.  Targeted Busulfan therapy with a steady-state concentration of 600-700 ng/mL in patients with sickle cell disease receiving HLA-identical sibling bone marrow transplant.

Authors:  S Maheshwari; A Kassim; R F Yeh; J Domm; C Calder; M Evans; B Manes; K Bruce; V Brown; R Ho; H Frangoul; E Yang
Journal:  Bone Marrow Transplant       Date:  2013-12-09       Impact factor: 5.483

7.  HLA Haploidentical Stem Cell Transplant with Pretransplant Immunosuppression for Patients with Sickle Cell Disease.

Authors:  Anna B Pawlowska; Jerry C Cheng; Nicole A Karras; Weili Sun; Leo D Wang; Alison D Bell; Lisa Gutierrez; Joseph Rosenthal
Journal:  Biol Blood Marrow Transplant       Date:  2017-09-20       Impact factor: 5.742

8.  Reduced toxicity, myeloablative HLA-haploidentical hematopoietic stem cell transplantation with post-transplantation cyclophosphamide for sickle cell disease.

Authors:  Volker Wiebking; Sebastian Hütker; Irene Schmid; Stefanie Immler; Tobias Feuchtinger; Michael H Albert
Journal:  Ann Hematol       Date:  2017-06-01       Impact factor: 3.673

9.  The ethics of a proposed study of hematopoietic stem cell transplant for children with "less severe" sickle cell disease.

Authors:  Robert S Nickel; Jeanne E Hendrickson; Ann E Haight
Journal:  Blood       Date:  2014-06-24       Impact factor: 22.113

10.  Pulmonary hypertension as a risk factor for death in patients with sickle cell disease.

Authors:  Mark T Gladwin; Vandana Sachdev; Maria L Jison; Yukitaka Shizukuda; Jonathan F Plehn; Karin Minter; Bernice Brown; Wynona A Coles; James S Nichols; Inez Ernst; Lori A Hunter; William C Blackwelder; Alan N Schechter; Griffin P Rodgers; Oswaldo Castro; Frederick P Ognibene
Journal:  N Engl J Med       Date:  2004-02-26       Impact factor: 91.245

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  3 in total

Review 1.  Evidence-Based Minireview: In young children with severe sickle cell disease, do the benefits of HLA-identical sibling donor HCT outweigh the risks?

Authors:  Niketa Shah; Lakshmanan Krishnamurti
Journal:  Hematology Am Soc Hematol Educ Program       Date:  2021-12-10

2.  Stable to improved cardiac and pulmonary function in children with high-risk sickle cell disease following haploidentical stem cell transplantation.

Authors:  Deborah Friedman; Allen J Dozor; Jordan Milner; Marise D'Souza; Julie-An Talano; Theodore B Moore; Shalini Shenoy; Qiuhu Shi; Mark C Walters; Elliott Vichinsky; Susan K Parsons; Suzanne Braniecki; Chitti R Moorthy; Janet Ayello; Allyson Flower; Erin Morris; Harshini Mahanti; Sandra Fabricatore; Liana Klejmont; Carmella van de Ven; Lee Ann Baxter-Lowe; Mitchell S Cairo
Journal:  Bone Marrow Transplant       Date:  2021-05-06       Impact factor: 5.483

Review 3.  Building a Fit for Purpose Clinical Trials Infrastructure to Accelerate the Assessment of Novel Hematopoietic Cell Transplantation Strategies and Cellular Immunotherapies.

Authors:  Steven M Devine; Mary M Horowitz
Journal:  J Clin Oncol       Date:  2021-01-12       Impact factor: 44.544

  3 in total

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