Autoimmune cytopenias following allogeneic hematopoietic stem cell transplant in pediatric patients: Response to therapy and late effects.

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Tytuł:: Autoimmune cytopenias following allogeneic hematopoietic stem cell transplant in pediatric patients: Response to therapy and late effects.
Autorzy:: Koo J; Children's Hospital Colorado, Division of Pediatric Hematology/Oncology/Bone Marrow Transplant, Colorado, University of Colorado Anschutz Medical Campus, Aurora.
Giller RH; Children's Hospital Colorado, Division of Pediatric Hematology/Oncology/Bone Marrow Transplant, Colorado, University of Colorado Anschutz Medical Campus, Aurora.
Quinones R; Children's Hospital Colorado, Division of Pediatric Hematology/Oncology/Bone Marrow Transplant, Colorado, University of Colorado Anschutz Medical Campus, Aurora.
McKinney CM; Children's Hospital Colorado, Division of Pediatric Hematology/Oncology/Bone Marrow Transplant, Colorado, University of Colorado Anschutz Medical Campus, Aurora.
Verneris MR; Children's Hospital Colorado, Division of Pediatric Hematology/Oncology/Bone Marrow Transplant, Colorado, University of Colorado Anschutz Medical Campus, Aurora.
Knight-Perry J; Children's Hospital Colorado, Division of Pediatric Hematology/Oncology/Bone Marrow Transplant, Colorado, University of Colorado Anschutz Medical Campus, Aurora.
Źródło:: Pediatric blood & cancer [Pediatr Blood Cancer] 2020 Sep; Vol. 67 (9), pp. e28591. Date of Electronic Publication: 2020 Jul 13.
Typ publikacji:: Journal Article
Język:: English
Imprint Name(s):: Original Publication: Hoboken, N.J. : John Wiley, c 2004-
MeSH Terms:: Anemia, Hemolytic, Autoimmune/*mortality
Graft vs Host Disease/*mortality
Hematologic Neoplasms/*mortality
Hematopoietic Stem Cell Transplantation/*mortality
Adolescent ; Adult ; Anemia, Hemolytic, Autoimmune/etiology ; Anemia, Hemolytic, Autoimmune/pathology ; Case-Control Studies ; Child ; Child, Preschool ; Female ; Follow-Up Studies ; Graft vs Host Disease/etiology ; Graft vs Host Disease/pathology ; Hematologic Neoplasms/pathology ; Hematologic Neoplasms/therapy ; Hematopoietic Stem Cell Transplantation/adverse effects ; Humans ; Infant ; Male ; Prognosis ; Retrospective Studies ; Survival Rate ; Young Adult
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Contributed Indexing:: Keywords: allogeneic HSCT; autoimmune cytopenia; hypogammaglobulinemia; late effects
Entry Date(s):: Date Created: 20200714 Date Completed: 20201207 Latest Revision: 20201214
Update Code:: 20240105
DOI:: 10.1002/pbc.28591
PMID:: 32658382
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Background: Autoimmune cytopenias (AICs) are rare, but serious complications of allogeneic hematopoietic cell transplantation (allo-HSCT).
Procedure: We performed a case-control study using 20 pediatric AIC cases and 40 controls, matched by stem cell source and primary indication comparing clinical and transplant characteristics, treatment, outcomes, and late effects.
Results: Cases were more likely to be human leukocyte antigen mismatched (P = 0.04). There was no difference in conditioning regimen, serotherapy use, graft-versus-host disease (GVHD) prophylaxis, incidence of acute or chronic GVHD, ABO compatibility, infections, and donor engraftment. The median time to AIC onset was 219 days (range, 97-1205 days) and AIC resolution was 365 days (range, 10 days to 2737.5 days). First-line therapies for AIC patients most commonly included corticosteroids (75%) and rituximab (55%). Only 25% of patients responded to first-line treatment. At a median of 611.5 days from last rituximab dose, 82.5% patients were still receiving intravenous immune globulin for hypogammaglobulinemia compared with 2.5% of controls (P < 0.0001). Iron overload was higher in AIC patients (P = 0.0004), as was avascular necrosis (P = 0.04). There was no difference in overall survival at one year after HSCT (85% vs 82.5%). Two patients with refractory autoimmune hemolytic anemia responded to daratumumab and had resolution of B-cell aplasia.
Conclusions: In this study, we find poor initial responses to AIC-directed therapies and significant late effects.
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