Refractory postallogeneic stem cell transplant pure red cell aplasia in remission after treatment with daratumumab

Bathini, Srilakshmi; Holtzman, Noa G.; Koka, Rima; Singh, Zeba N.; Wilding, Emily; Zou, Ying; Ruehle, Kathleen; Kocoglu, Mehmet H.; Badros, Ashraf; Hardy, Nancy M.; Yared, Jean A.; Rapoport, Aaron P.; Fontaine, Magali J.; Emadi, Ashkan; Chaer, Firas El; Dahiya, Saurabh

doi:10.1002/ajh.25515

Cited by 30 publications

(28 citation statements)

References 6 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…This is consistent with a previous case report where daratumumab achieved a sustained remission in an infant with both AIHA and ITP who had previously failed multiple lines of therapy. 33,[38][39][40][41][42] Although reports agree that there is efficient periph-…”

Section: Discussionmentioning

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

View full text Add to dashboard Cite

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.

show abstract

Section: Discussionmentioning

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

View full text Add to dashboard Cite

show abstract

“…Recently, treatment with daratumumab for post‐HSCT AIC was reported in three patients, two of whom were cured . Additionally, two case reports of successful treatment with daratumumab for delayed red cell engraftment following HSCT were recently published . No significant adverse effects were noted in these cases except for delayed recovery of humoral immunity necessitating substitution with IVIG.…”

Section: Discussionmentioning

confidence: 99%

“…Recently, there have been reports of successful treatment of resistant post-HSCT AIHA and of post-HSCT pure red cell aplasia with daratumumab. [6][7][8] We present here a case of a pediatric patient with primary myelofibrosis of infancy caused by VPS45 protein deficiency, who developed severe refractory post-HSCT hemolytic anemia and thrombocytopenia and was successfully treated by daratumumab with complete resolution of symptoms and normalization of blood counts.…”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Successful treatment with daratumumab for post‐HSCT refractory hemolytic anemia

Even‐Or

NaserEddin

Shadur

et al. 2019

Pediatric Blood & Cancer

View full text Add to dashboard Cite

Autoimmune cytopenias (AIC) following allogeneic hematopoietic stem cell transplantation (HSCT) may cause significant morbidity and mortality and are often challenging to treat. We present a case of a pediatric patient with primary myelofibrosis of infancy caused by VPS45 protein deficiency, who developed severe refractory hemolytic anemia and immune‐mediated thrombocytopenia 3.5 months following HSCT. After the failure of several treatments, he received daratumumab, an anti‐CD38 specific antibody, and demonstrated fast and sustained response. The only side effect was delayed recovery of humoral immunity. Daratumumab, by targeting antibody‐producing plasma cells, may be a valid treatment option for refractory post‐HSCT AIC.

show abstract

“…Recent case reports have described the efficacy of daratumumab to treat refractory pure red cell aplasia (PRCA) following major ABO mismatched allogeneic hematopoietic stem cell transplantation (HSCT). [1][2][3][4] In this report, we describe the use of daratumumab as a first-line agent for treatment of delayed red blood cell (RBC) engraftment in the setting of major ABO mismatched pediatric HSCT and provide a review of the literature.…”

Section: Introductionmentioning

confidence: 99%

Daratumumab for delayed RBC engraftment following major ABO mismatched haploidentical bone marrow transplantation

et al. 2021

View full text Add to dashboard Cite

Background Recent case reports have described the efficacy of daratumumab to treat refractory pure red cell aplasia (PRCA) following major ABO mismatched allogeneic hematopoietic stem cell transplantation (HSCT). In this report, we describe the use of daratumumab as a first‐line agent for treatment of delayed red blood cell (RBC) engraftment following a major ABO mismatched pediatric HSCT and provide a review of the literature. Study Design and Materials We report on a 14‐year‐old with DOCK8 deficiency who underwent a myeloablative, haploidentical bone marrow transplant from her major ABO mismatched sister (recipient O+, donor A+) for treatment of her primary immunodeficiency. Despite achieving full donor chimerism, she had delayed RBC engraftment requiring ongoing transfusions. Due to iron deposition, symptomatic anemia, and persistence of anti‐A iso‐hemagglutinins despite discontinuation of immunosuppression, treatment for delayed RBC engraftment with the CD38‐targeted monoclonal antibody daratumumab was selected as a less immunosuppressive agent that could more selectively target iso‐hemagglutinin producing plasma cells without causing broad B‐cell aplasia. Results Clinical effect with daratumumab was demonstrated by reduced iso‐hemagglutinin titer, increased reticulocytosis, normalization of her hemoglobin, and transfusion independence. In the 11‐month follow‐up period to date, no additional transfusions or immunosuppression have been necessary, despite persistence of low‐level anti‐A iso‐hemagglutinin. Conclusion Our experience suggests that daratumumab was an effective first‐line therapy for delayed RBC engraftment and that earlier consideration for daratumumab in treatment of delayed RBC engraftment may be warranted.

show abstract

Refractory postallogeneic stem cell transplant pure red cell aplasia in remission after treatment with daratumumab

Cited by 30 publications

References 6 publications

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

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

Successful treatment with daratumumab for post‐HSCT refractory hemolytic anemia

Daratumumab for delayed RBC engraftment following major ABO mismatched haploidentical bone marrow transplantation

Contact Info

Product

Resources

About