Survival and toxicity outcomes of hematopoietic stem cell transplantation for pediatric patients with Fanconi anemia: a unified multicentric national study from the Spanish Working Group for Bone Marrow Transplantation in Children.
Laura MurilloM González-VicentB Argilés AparicioI Badell SerraA Rodríguez VillaM L Uria OficialdeguiM López-DuarteC Beléndez-BielerA Sastre UrgellesJ Sevilla NavarroChristina Diaz de Heredianull nullPublished in: Bone marrow transplantation (2020)
Hematopoietic stem cell transplantation (HSCT) is currently the only curative option for hematological manifestations in patients with Fanconi anemia (FA). We report the outcome of 34 patients with FA inside a collaborative multicenter national study based on recommendations of Spanish Working Group for Bone Marrow Transplantation in Children (GETMON) between 2009 and 2016. Fludarabine-based conditioning regimen was carried out in all patients, with low dose total body irradiation in unrelated transplants. Disease status before HSCT was bone marrow failure (BMF) in 30 patients and myelodysplastic syndrome (MDS) in four. Donors were matched siblings donors (MSD) in 18, matched unrelated donors (MUD) in 15, and one haploidentical donor. All except one patient engrafted. Cumulative incidence of grades II-IV acute graft-versus-host disease (GVHD) was 29% and 11% for chronic GVHD. Median follow-up after HSCT was 6.5 years. Seven patients (21%) died due to transplant-related causes, two (6%) because of MDS relapse, and one (3%) after a squamous cell carcinoma. Overall survival (OS) was 73% at 5 years post-transplant, with no differences between MSD and MUD transplants. OS for patients with BMF was 80% while for MDS was 25%. Our data suggest HSCT can cure hematologic manifestations of most FA patients with BMF.
Keyphrases
- bone marrow
- end stage renal disease
- chronic kidney disease
- squamous cell carcinoma
- low dose
- newly diagnosed
- ejection fraction
- prognostic factors
- quality improvement
- stem cells
- young adults
- peritoneal dialysis
- oxidative stress
- patient reported outcomes
- risk factors
- liver failure
- kidney transplantation
- case report
- clinical trial
- drug induced
- adipose tissue
- respiratory failure
- stem cell transplantation
- cell therapy
- acute lymphoblastic leukemia
- acute respiratory distress syndrome
- lymph node metastasis
- radiation therapy
- cross sectional
- extracorporeal membrane oxygenation
- big data
- deep learning