First-Line Immunosuppressive Treatment in Children with Aplastic Anemia: Rabbit Antithymocyte Globulin

Pawelec, Katarzyna; Salamonowicz, Małgorzata; Panasiuk, Anna; Demkow, Urszula; Kowalczyk, Jerzy; Balwierz, Walentyna; Zaleska-Czepko, Ewa; Chybicka, Alicja; Szmyd, Krzysztof; Szczepański, Tomasz; Bubała, H; Wysocki, Mariusz; Kurylak, Andrzej; Wachowiak, Jacek; Szpecht, Dawid; Młynarski, Wojciech; Bulas, Monika; Krawczuk‐Rybak, Maryna; Leszczyńska, Elżbieta; Urasiński, Tomasz; Peregud-Pogorzelski, Jarosław; Balcerska, Anna; Kaczorowska-Hac, Barbara; Matysiak, Michał

doi:10.1007/5584_2014_38

Cited by 9 publications

(5 citation statements)

References 24 publications

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“…26 Another study included 63 pediatric SAA patients without MSD who were treated with rabbit ATG and CsA as firstline IST. 27 Thirty-four of these patients (64.9%) went into remission, but only 24 (38.0%) went into CR. The 10-year OS and FFS were 67 and 57%.…”

Section: Discussionmentioning

confidence: 96%

Haploidentical transplantation for pediatric patients with acquired severe aplastic anemia

Zhang

Wang

et al. 2016

Bone Marrow Transplant

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Techniques for haploidentical hematopoietic stem cell transplantation (haplo-HSCT) to treat severe aplastic anemia (SAA) have recently improved, but no protocol has been evaluated in a large number of pediatric patients. Fifty-two children with SAA received haplo-HSCT in our center. The treatment protocol used G-CSF-primed bone marrow with G-CSF-mobilized PBSCs without in vitro T-cell depletion. The conditioning regimen included busulfan/cyclophosphamide and antithymocyte globulin. Fifty-one patients achieved primary engraftment; one child died of regimen-related toxicity on the day +1. Secondary graft failure occurred in three patients. The cumulative incidences of aGVHD grade II-IV and grade III-IV were 39.2±0.5 and 13.7±0.2%, respectively. The cumulative incidence of cGVHD was 34.2±0.5%. The 3-year overall and failure-free survival rates were 84.5±5.0 and 82.7±5.2%, respectively, with a median follow-up time of 744.5 days (100-3294) for surviving patients. The Eastern Cooperative Oncology Group score was the only predictor of overall and failure-free survival rates. Clinical outcomes were similar between the upfront and salvage group. This result suggests that both newly diagnosed and refractory pediatric SAA patients benefit from haplo-HSCT, especially when patients are in good general condition. Therefore, haplo-HSCT might be an alternative therapy for pediatric SAA patients without HLA-matched sibling donors.

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Section: Discussionmentioning

confidence: 96%

Haploidentical transplantation for pediatric patients with acquired severe aplastic anemia

Zhang

Wang

et al. 2016

Bone Marrow Transplant

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“…In contrast, the EWOG-SAA-2010 interim analysis showed a lower overall response rate (ORR) to IST in patients treated with rATG compared with hATG (22% vs 42%, P = 0.03) but without differences in OS (88% vs 93%) (76). Similarly, Pawelec et al demonstrated the 3-year OS of 78% and the 10-year survival of 67% for hATG and rATG, respectively (77). However, in a meta-analysis by Hayakawa et al, the ORR was significantly higher for hATG vs rATG (risk ratio [RR], 1.27; 95% CI, 1.05-1.54; P = 0.015) (78).…”

Section: Evaluation Of Pediatric Recommendationsmentioning

confidence: 94%

The state of the art in the treatment of severe aplastic anemia: immunotherapy and hematopoietic cell transplantation in children and adults

Piekarska,

Pawelec,

Szmigielska-Kapłon

et al. 2024

Front. Immunol.

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Acquired aplastic anemia (AA) is an immune-mediated bone marrow (BM) failure where marrow disruption is driven by a cytotoxic T-cell–mediated autoimmune attack against hematopoietic stem cells. The key diagnostic challenge in children, but also in adults, is to exclude the possible underlying congenital condition and myelodysplasia. The choice of treatment options, either allogeneic hematopoietic cell transplantation (alloHCT) or immunosuppressive therapy (IST), depends on the patient’s age, comorbidities, and access to a suitable donor and effective therapeutic agents. Since 2022, horse antithymocyte globulin (hATG) has been available again in Europe and is recommended for IST as a more effective option than rabbit ATG. Therefore, an update on immunosuppressive strategies is warranted. Despite an improved response to the new immunosuppression protocols with hATG and eltrombopag, some patients are not cured or remain at risk of aplasia relapse or clonal evolution and require postponed alloHCT. The transplantation field has evolved, becoming safer and more accessible. Upfront alloHCT from unrelated donors is becoming a tempting option. With the use of posttransplant cyclophosphamide, haploidentical HCT offers promising outcomes also in AA. In this paper, we present the state of the art in the management of severe AA for pediatric and adult patients based on the available guidelines and recently published studies.

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“…Immunosuppressive therapy, including cyclosporin and antithymocyte globulin is used in patients for whom bone marrow transplantation is not an option, with response rates of 60-80 % (Pawelec et al 2008(Pawelec et al , 2015Chang et al 2010;Deyell et al 2011;Bagby et al 2004). However, supportive care with red blood cell transfusions is also essential in many patients to maintain adequate hemoglobin level (70-80 g/L), particularly since the response to immunosuppressive therapy may be delayed.…”

Section: Introductionmentioning

confidence: 98%

Influence of Iron Overload on Immunosuppressive Therapy in Children with Severe Aplastic Anemia

Pawelec

Salamonowicz

Panasiuk

et al. 2015

Advances in Experimental Medicine and Biology

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Children with severe aplastic anemia (AA) require multiple transfusions of the red blood cells during the immunosuppressive therapy. This leads to iron overload and manifests as elevated levels of ferritin in blood. The aim of this study was a retrospective analysis of the influence of the elevated serum ferritin on the overall survival, event-free survival, the risk of relapse, and response to treatment in children with AA during immunosuppressive therapy. We analyzed 38 children with AA (19 girls, 19 boys, aged 2-17 years) treated according to the obligatory protocol for AA in Poland. The response rate was assessed on days 84, 112, and 360. Patients were divided into three groups: group I consisted of children with ferritin below 285 ng/mL (6 children), group II with ferritin between 286 and 1,000 ng/mL (13 children), and group III ferritin>1,000 ng/mL (19 children). Kaplan-Meier plot was used to estimate the overall survival and event-free survival. We found the overall survival did not differ between the three groups. Event-free survival was significantly shorter (p=0.03) in patients with ferritin levels>1,000 ng/mL compared with the groups with ferritin bellow 1,000 ng/mL. The time to relapse was significantly shorter in group III than in the other two groups (p=0.02). We also found the differences in the treatment response at day 84 (p=0.03) and day 112 (p<0.0001) of immunosuppressive therapy. These findings confirm a negative influence of iron overload in children with AA on the effect of treatment and the risk of relapse.

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First-Line Immunosuppressive Treatment in Children with Aplastic Anemia: Rabbit Antithymocyte Globulin

Cited by 9 publications

References 24 publications

Haploidentical transplantation for pediatric patients with acquired severe aplastic anemia

Haploidentical transplantation for pediatric patients with acquired severe aplastic anemia

The state of the art in the treatment of severe aplastic anemia: immunotherapy and hematopoietic cell transplantation in children and adults

Influence of Iron Overload on Immunosuppressive Therapy in Children with Severe Aplastic Anemia

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