Reduced-intensity conditioning and stem cell transplantation in infants with Diamond Blackfan anemia

Crazzolara, Roman; Kropshofer, Gabriele; Haas, Oskar A.; Matthes‐Martin, Susanne; Kager, Leo

doi:10.3324/haematol.2016.157149

Cited by 15 publications

(10 citation statements)

References 15 publications

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“…To date, reduced-intensity regimens have been successfully applied in a small number of selected DBA patients. 20,21 For example, in their paper, Crazzolara et al reported 3 cases of infants of 7, 9, and 13 months successfully transplanted after either a thiotepafludarabine or thiotepa-treosulfan-fludarabine conditioning regimen. These infants had a low transfusion burden that may play a role in the success of HSCT.…”

Section: Discussionmentioning

confidence: 99%

Favorable outcomes of hematopoietic stem cell transplantation in children and adolescents with Diamond-Blackfan anemia

et al. 2020

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Diamond-Blackfan anemia (DBA) is a congenital pure red cell aplasia associated with congenital abnormalities and cancer predisposition. Allogeneic hematopoietic stem cell transplantation (HSCT) can correct the hematological phenotype and is indicated in transfusion-dependent patients. In 70 children reported to the German DBA and French HSCT registries, HSCT was performed from 1985 to 2017. Median age at HSCT was 5.5 years (range, 0.9-17.3 years). Two-thirds of patients (64%) were transplanted from a matched sibling donor (MSD), and most procedures were performed after the year 1999 (73%). Primary engraftment was achieved in all patients. One patient developed secondary graft failure. Cumulative incidence of acute graft-versus-host disease (GVHD) was 24% for °II-IV (95% confidence interval [CI], 16% to 37%) and 7% for °III-IV (95% CI, 3% to 17%); cumulative incidence of chronic GVHD was 11% (95% CI, 5% to 22%). The probability of chronic GVHD-free survival (cGFS) was 87% (95% CI, 79% to 95%) and significantly improved over time (<2000: 68% [95% CI, 47% to 89%] vs ≥2000: 94% [95% CI, 87% to 100%], P < .01). cGFS was comparable following HSCT from a MSD and an unrelated donor (UD). Of note, no severe chronic GVHD or deaths were reported following MSD-HSCT after 1999. The difference of cGFS in children transplanted <10 years of age compared with older patients did not reach statistical significance (<10 years: 90% [95% CI, 81% to 99%] vs 10-18 years 78% [95% CI, 58% to 98%]). In summary, these data indicate that HSCT is efficient and safe in young DBA patients and should be considered if a MSD or matched UD is available. HSCT for transfusion dependency only must be critically discussed in older patients.

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Section: Discussionmentioning

confidence: 99%

Favorable outcomes of hematopoietic stem cell transplantation in children and adolescents with Diamond-Blackfan anemia

et al. 2020

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“…Limitations of our case series include its retrospective, single‐institution design and heterogeneity of both inherited and acquired BMFS diagnoses which limits comparisons to single diagnosis studies, including studies of RIC for pediatric patients with SAA, 6 DBA, 7 and PNH with SAA 8 . However, the heterogeneity of diagnoses—including a cohort of four unrelated patients with CAMT—also makes our excellent outcomes broadly generalizable to pediatric patients with BMFS (with the exclusion of patients with marrow failure due to Fanconi anemia, dyskeratosis congenita, or Shwachman‐Diamond syndrome—all notably absent from our case series).…”

Section: Discussionmentioning

confidence: 99%

Irradiation‐free RIC HSCT has a tolerable safety profile and is effective therapy for pediatric bone marrow failure syndromes

Wang

Anderson

Gloude

et al. 2020

Pediatric Transplantation

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For patients with bone marrow failure syndromes (BMFS) who may tolerate gradual donor engraftment and achieve adequate disease control with stable mixed chimerism, RIC regimens may be preferable to myeloablative regimens. We performed a retrospective analysis of outcomes for patients who underwent HSCT at our institution between 2009 and 2017 for BMFS using an irradiation‐free RIC regimen. Fourteen pediatric patients with BMFS received fludarabine (30 mg/m2 IV daily × 3), thiotepa (5 mg/kg IV every 12 hours × 2), and melphalan (70 mg/m2 IV daily × 2) prior to HSCT. Our cohort included the following diagnoses: SAA (n = 7), CAMT (n = 4), SCN (n = 1), DBA (n = 1), and non‐Fanconi congenital BMF (n = 1). Seven patients underwent a MSD transplant; seven underwent an unrelated donor transplant. All patients are alive with median follow‐up of 1112 days (range 455‐2549 days). The median time to neutrophil engraftment was 16 days (range 10‐26 days). All were transfusion independent by day + 100. The highest grade of aGVHD was grade 2; 8 (57%) did not develop aGVHD. Four (28.5%) developed extensive cGVHD, 4 (28.5%) developed limited cGVHD, and 6 (43%) did not develop cGVHD. No patients developed SOS. None died from GVHD or infectious complications. HSCT with RIC with fludarabine, thiotepa, and melphalan for BMFS was effective with a tolerable safety profile. Probability of OS at 100 days and 1 year was 100%.

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“…Secondary graft failure, n (%) 3 7 young patients at risk of severe complications and in those with high iron overload. 31,32 Graft failure is a matter of great concern in HCT. In our study, primary GF was observed in 5 out of 44 patients (11%), data similar to that has been observed in other studies.…”

Section: Discussionmentioning

confidence: 99%

Hematopoietic cell transplantation for Diamond Blackfan anemia: A report from the Pediatric Group of the Brazilian Bone Marrow Transplantation Society

Darrigo

Loth

Kuwahara

et al. 2020

European J of Haematology

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Objectives The aim of this study was to analyze the outcomes of children with Diamond‐Blackfan anemia (DBA) treated in Brazil with hematopoietic cell transplantation (HCT). Methods We performed a retrospective analysis of 44 pediatrics patients transplanted between 1990 and 2018. The median age of patients was 5 years, and 57% were male. Twenty‐five received their first HCT from an HLA‐matched sibling donor (MSD), 12 from a HLA matched unrelated bone marrow donor (MUD 10/10, n = 12) and 7 other HLA mismatched donors (MMD). Results After a median follow‐up of 4 years, estimate 5‐year overall survival (OS) for the entire cohort was 70%, 80% for MSD group, 73% for MUD, and 29% for MMD. Thirty‐eight out of the 44 evaluable patients engrafted successfully. Primary and secondary graft failure was observed in five and three patients, respectively. Rates of grade II‐IV and III‐IV acute graft‐versus‐host disease (aGVHD) were 25% and 18%, respectively. Nine patients developed chronic GVHD (cGVHD). Conclusion Overall survival rates observed after HLA matched donors transplant for DBA were comparable to those reported from higher‐income countries and international registries.

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Reduced-intensity conditioning and stem cell transplantation in infants with Diamond Blackfan anemia

Cited by 15 publications

References 15 publications

Favorable outcomes of hematopoietic stem cell transplantation in children and adolescents with Diamond-Blackfan anemia

Favorable outcomes of hematopoietic stem cell transplantation in children and adolescents with Diamond-Blackfan anemia

Irradiation‐free RIC HSCT has a tolerable safety profile and is effective therapy for pediatric bone marrow failure syndromes

Hematopoietic cell transplantation for Diamond Blackfan anemia: A report from the Pediatric Group of the Brazilian Bone Marrow Transplantation Society

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