Top Back to top

Summary of recent EBMT publications


Pregnancy and pregnancy outcomes after hematopoietic stem cell transplantation in childhood: a cross-sectional survey of the EBMT Pediatric Diseases Working Party

Diesch-Furlanetto T et al

Hum Reprod . 2021 Oct 18;36(11):2871-2882. doi: 10.1093/humrep/deab199.

One of the remaining unresolved major transplant related morbidities is loss of fertility, most detrimental in children, adolescents and young adults with non-malignant diseases.

Preparative regimens of HCT remain highly gonadotoxic, which leads to gonadal failure and pubertal development disorders. There are few population-based studies assessing the risk of future infertility in children after HCT.

Diesch-Furlanetto T et al conducted a retrospective study to investigate natural or assisted conceptions and their outcomes in patients <18 years old before their first transplantation who received HCT between 1995 and 2016, based on entries the EBMT registry.

Registry data combined with focused questionnaires. 62 988 pediatric patients received a first HCT in EBMT centers between 1995 and 2016. Pregnancy was reported in 406 patients. Details concerning the first pregnancy and pregnancy outcome were obtained from 99 patients (24%) from the returned questionnaires.

One major outcome was that compared with healthy women at their first child's birth (29 years old), the transplanted women delivered 5 years earlier.

In terms of conception modality, 13/25 (52%) females conditioned with total body irradiation (TBI) and 50/52 (96%) of those conditioned without TBI conceived naturally.

In the females, 63/70 (90%) of all conceptions resulted in a live birth, 49/63 (84.5%) were at term and 43/46 (93%) had normal birthweight.

These results are encouraging and showed that natural conception is possible. Reproductive health surveillance and all efforts of fertility preservation in younger transplanted patients should be undertaken.

EBMT/ESID inborn errors working party guidelines for hematopoietic stem cell transplantation for inborn errors of immunity

Lankester AC et al

Bone Marrow Transplantation (2021) 56:20522062;

Inborn errors of immunity (IEI) are a group of rare heterogeneous diseases. More than 400 monogenetic IEI have been identified and a genetic diagnosis can be made in many patients with a presumed immune deficiency disorder.

Therefore, it is pivotal to follow a homogenous approach in order to gain some evidence in these rare diseases.

In that regard the new guidelines for hematopoietic stem cell transplantation for inborn errors of immunity are a recurring and awaited highlight for those involved in the care of patients with IEI.

Comparison of mycophenolate mofetil and calcineurin inhibitor versus calcineurin inhibitor-based graft-versus-host-disease prophylaxis for matched unrelated donor transplant in acute myeloid leukemia. A study from the ALWP of the EBMT

Paviglianti A et al

Bone Marrow Transplant. 2021 May;56(5):1077-1085. doi: 10.1038/s41409-020-01155-z. Epub 2020 Nov 28.

Paviglianti et al investigated in a prospective randomized trial the impact of the addition of MMF to a CsA based RIC regimen.

They analyzed 497 patients with AML in complete remission (CR) who underwent matched unrelated donor (MUD) hematopoietic stem cell transplantation (HSCT). All patients received a fludarabine busulfan RIC regimen and anti-thymocyte globulin (ATG) with either CsA alone or in combination with MMF.

The cumulative incidence (CI) of grade II-IV acute GvHD was 27% for CsA and 33% for CsA+MMF (p = 0.25). The 2-year CI of chronic GvHD was 38% and 33% for the CsA and the CsA+MMF group, respectively (p = 0.26). On multivariate analysis, no statistically significant differences with respect to relapse incidence (RI), non-relapse mortality (NRM), leukemia-free survival (LFS), overall survival (OS), acute and chronic GvHD were found between the two groups, including a subgroup analysis in peripheral blood stem cells (PBSC) recipients.

These data are an important and unexpected piece of information for this subset of MUD HSCT with AML and a RIC conditioning. Inasmuch they can be extrapolated to other entities, including non-malignant diseases and children, where other outcome parameter such as graft rejection and GvHD under the circumstances of an immature immune system may play into the picture, needs further studies.

The most evident result is that nothing can replace a hypothesis driven, well controlled prospective trial. Many more are urgently needed in our field.