Treatment‐refractory multi‐lineage autoimmune cytopenia after unrelated cord blood transplantation: Remission after combined bortezomib and vincristine treatment

Waespe, Nicolas; Zeilhofer, Ulrike B.; Güngör, Tayfun

doi:10.1002/pbc.25122

Cited by 16 publications

(11 citation statements)

References 20 publications

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“…Bortezomib. Bortezomib is an inhibitor of 26S proteasome that has been successfully used in AC following SCT (Waespe et al, 2014) and in a case of IgM-driven AIHA secondary to a monoclonal gammopathy (Carson et al, 2010). Seven children with primary, secondary or post-transplant AC, including one with ES, treated with bortezomib, have also been recently reported (Khandelwal et al, 2014) and a clinical trial is on-going in the setting of post-transplant AC (ClinicalTrials.gov identifier: NCT01930253).…”

Section: New Drugsmentioning

confidence: 99%

How I manage Evans Syndrome and AIHA cases in children

Miano

2015

Br J Haematol

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The management of Evans Syndrome in children is challenging due to the lack of evidence-based data on treatment. Steroids, the first-choice therapy, are successful in about 80% of cases. For children who are resistant, relapse or become steroid-dependent, rituximab is considered a valid second-line treatment, with the exception of those with an underlying diagnosis of autoimmune lymphoproliferative syndrome who may benefit from other options such as mycophenolate mofetil and sirolimus. Better knowledge of the immunological mechanisms underlying cytopenias and the availability of new immunosuppressive drugs can be helpful in the choice of more targeted therapies that would enable the reduction of the use of long-term steroid administration or other more aggressive options, such as splenectomy or stem cell transplantation. This manuscript provides an overview of the pathogenic background of the disease, and suggests a clinical approach to diagnosis and treatment with a particular focus on the management of relapsing/resistant disease.

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Section: New Drugsmentioning

confidence: 99%

How I manage Evans Syndrome and AIHA cases in children

Miano

2015

Br J Haematol

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“…5,6 Prior studies have identified several risk factors for AIC development including nonmalignant transplant indications, chemotherapy naivety, unrelated donors, cord blood stem cell source, graft-vs-host disease (GVHD), reduced-intensity preparative regimens, serotherapy, and cytomegalovirus (CMV) reactivation. [7][8][9][10] Corticosteroids and rituximab are the most common first-line therapies for AICs. 11 However, these interventions have side effects and long-term consequences.…”

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confidence: 99%

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

Koo

Giller

Quinones

et al. 2020

Pediatric Blood & Cancer

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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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“…11,12 Furthermore, although infrequent, a CBT may be a risk factor for autoimmune cytopenia. 4,13,14 Because a 7/8 HLA allelic-matched sibling was born, CBT emerged as a therapeutic option for the cure of immunosuppressive-therapy-resistant MDS in our case. However, if we consider the risk of relapse, infection or autoimmune cytopenia, HLA-haploidentical BM may become a higher priority source compared with CB in the treatment of pediatric MDS patients without siblings or HLA-matched unrelated donors.…”

mentioning

confidence: 91%

“…Recently, several case reports have suggested that bortezomib, a 26 S proteasome inhibitor that eliminates plasma cells and decreases the production of IgG antibodies, was safe and effective for autoimmune cytopenia after HSCT in both adults and children. [3][4][5] From day +255, 1.3 mg/m 2 of bortezomib was intravenously administered per week for 4 weeks in combination with rituximab. A continuous ANC of 40.5 × 10 9 /L was observed from day +300.…”

mentioning

confidence: 99%