C. Bonfim1,2,3, S. Nichele1,2, G. Loth1,3, A. Koliski1, L. Ribeiro1, R.A. Toassa Gomes Mosquer1, C.C.

Kuwahara Dumke3, F. Moreira de Lara Benini3, P.R. Dorini Pelegrina3, A. Mello Rodrigues1,3, C.
Martins de Almeida Peixoto3, J.L. Mello Bach3, J.P. Trennepohl1,2, L.M. Miranda de Gouvêa2, R.
Muratori1, D. Vandresen Pilonetto1, J. Folloni Fernandes4,5, C. Arrais6, A. Cardoso Martins Lima1, R.
Pasquini1,2
1
Hospital de Clínicas da Universidade Federal do Paraná, Curitiba, Brazil, 2Hospital Nossa Senhora
das Graças, Curitiba, Brazil, 3Hospital Infantil Pequeno Príncipe, Curitiba, Brazil, 4Instituto do
Tratamento do Câncer Infantil – Instituto da Criança – HCFMUSP, São Paulo, Brazil, 5Hospital Albert
Einstein, São Paulo, Brazil, 6Hospital São Paulo – Escola Paulista de Medicina, São Paulo, Brazil

Background: Fanconi anemia (FA) is the most common inherited bone marrow failure in childhood.
Hematopoietic cell transplantation (HCT) can cure the hematological complications related to the
disease with better results when patients are transplanted at a young age, in the aplastic phase, and
with a matched related or unrelated donor. When a matched donor is not available, the use of
haploidentical donor or unrelated cord blood grafts can be an alternative. We evaluated the
outcomes of FA patients receiving haploidentical transplantation using post-transplantation
cyclophosphamide and serotherapy (HAPLO-PTCY-serotherapy) compared with a historical cohort
of patients who underwent unrelated cord blood transplantation (UCBT).
Methods: A retrospective, longitudinal, non-randomized, observational study including 89 patients
with FA transplanted in 3 HCT centers. Database and records were revised, and statistics were
performed using the EZR program. Sample: UCBT cohort: 42 patients at a median age of 8 years (3-
19) transplanted between 2000 and 2011. Only 2 patients were older than 16 years. The majority
had transfusion-dependent aplastic anemia (n=41) and received a fludarabine-based regimen
(n=38). GVHD prophylaxis consisted of cyclosporine (CsA) and steroids in 57%. HLA compatibility:
6/6 (n=5); 5/6 (n=16) and 4/6 (n=21). Haplo-PTCY-serotherapy cohort: 47 patients at a median age
of 10 years (4-45) transplanted between 2013 and 2020. 83% had transfusion-dependent aplastic
anemia and 12 were older than 16 years. All received bone marrow from haploidentical donors and a
modified reduced-intensity protocol with Fludarabine, TBI 200 cGys, Serotherapy (ATG/Campath),
and PTCY 50-60 mg/kg + CsA + MMF.
Results: When compared to the UCBT cohort, the Haplo-PTCY-serotherapy platform had a much
lower rejection rate at 100-days (6% vs 31%, p=0.003). The cumulative incidence (CI) of D100 acute
grade II-IV GVHD was similar (HAPLO: 28% vs UCBT:  19%; p=0.13), but the 2-year CI of chronic
GVHD was significantly higher (HAPLO: 48% vs UCBT: 19%; p=0.003). CMV reactivation and
hemorrhagic cystitis at D100 were also much higher after HAPLO-PTCY than UCBT (79% vs 38%;
p=0.0001) and (55% vs 9%; p=0.0001) respectively. Transplant-related mortality (TRM) at 180 days
was higher in the UCBT group (55% vs 6%; p=0.0001). Overall, 2-year survival was significantly
improved after haplo-PTCY when compared to the historical UCBT group (82% vs 36%; p=0.0001),
even though the HAPLO-PTCY cohort also included adult patients as well as patients with advanced
disease (eleven out of 12 older patients and 6 out of 8 patients with advanced disease are alive and
engrafted).
Conclusions: Compared to the historical UCBT cohort, the haplo-PTCY-serotherapy platform
significantly increased the survival in Fanconi Anemia by decreasing the rejection rate and TRM.
This excellent survival came with the high cost of viral reactivation and GVHD, suggesting new
strategies are needed to prevent and treat these complications.
Disclosure: Nothing to declare

Wednesday, March 23, 09:54 - 10:03

OS11 Oral session 11: Aplastic anemia, autoimmune diseases