Choice of conditioning regimens for bone marrow transplantation in severe aplastic anemia

Bejanyan, Nelli; Kim, Soyoung; Hebert, Kyle; Kekre, Natasha; Abdel‐Azim, Hisham; Ahmed, Ibrahim; Aljurf, Mahmoud; Badawy, Sherif M.; Beitinjaneh, Amer; Boelens, Jaap Jan; Díaz, Miguel Ángel; Dvorak, Christopher C.; Gadalla, Shahinaz M.; Gajewski, James; Gale, Robert Peter; Ganguly, Siddhartha; Gennery, Andrew R.; George, Biju; Gergis, Usama; Gómez‐Almaguer, David; González-Vicent, Marta; Hashem, Hasan; Kamble, Rammurti T.; Kasow, Kimberly A.; Lazarus, Hillard M.; Mathews, Vikram; Orchard, Paul J.; Pulsipher, Michael A.; Ringdén, Olle; Schultz, Kirk R.; Teira, Pierre; Woolfrey, Ann E.; Saldaña, Blachy J. Dávila; Savani, Bipin N.; Winiarski, Jacek; Yared, Jean A.; Weisdorf, Daniel J.; Antin, Joseph H.; Eapen, Mary

doi:10.1182/bloodadvances.2019000722

Cited by 47 publications

(32 citation statements)

References 28 publications

(56 reference statements)

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“…Transplantation conditioning regimens for patients with severe aplastic anemia vary by the type of donor [19]. Other reports have shown an effect of conditioning regimen for survival after HLA-matched sibling transplants [19]. None of the patients in the present analysis received cyclophosphamide alone or with fludarabine-conditioning regimens associated with higher graft failure and mortality rates [19].…”

Section: Discussionmentioning

confidence: 73%

“…Other reports have shown an effect of conditioning regimen for survival after HLA-matched sibling transplants [19]. None of the patients in the present analysis received cyclophosphamide alone or with fludarabine-conditioning regimens associated with higher graft failure and mortality rates [19]. The cell dose of the graft also has been associated with graft failure; it is recommended that bone marrow grafts contain a minimum of 3 £ 10 8 /kg TNCs to avoid graft failure [20].…”

Section: Discussionmentioning

confidence: 78%

Section: Discussionmentioning

confidence: 99%

“…HLA-matched sibling transplants were mostly offered within 6 months of diagnosis, and alternative donor transplants were offered later after failure of at least 1 course of immunosuppressive therapy [24]. Recipients of cryopreserved and noncryopreserved grafts were matched 19). Interval between diagnosis and HCT: z 77% HLA-matched sibling transplant; 23% HLA-matched or mismatched unrelated donor transplant.…”

Section: Discussionmentioning

confidence: 99%

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Hematopoietic Cell Transplantation with Cryopreserved Grafts for Severe Aplastic Anemia

Eapen¹,

Zhang²,

Tang³

et al. 2020

Biology of Blood and Marrow Transplantation

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With the COVID-19 pandemic and the ensuing barriers to the collection and transport of donor cells, it is often necessary to collect and cryopreserve grafts before initiation of transplantation conditioning. The effect on transplantation outcomes in nonmalignant disease is unknown. This analysis examined the effect of cryopreservation of related and unrelated donor grafts for transplantation for severe aplastic anemia in the United States during 2013 to 2019. Included are 52 recipients of cryopreserved grafts who were matched for age, donor type, and graft type to 194 recipients who received noncryopreserved grafts. Marginal Cox regression models were built to study the effect of cryopreservation and other risk factors associated with outcomes. We recorded higher 1-year rates of graft failure (hazard ratio [HR], 2.26; 95% confidence interval, 1.17 to 4.35; P = .01) and of 1-year overall mortality (HR, 3.13; 95% CI, 1.60 to 6.11; P = .0008) after transplantation of cryopreserved compared with noncryopreserved grafts, with adjustment for sex, performance score, comorbidity, cytomegalovirus serostatus, and ABO blood group match. The incidence of acute and chronic graft-versus-host disease did not differ between the 2 groups. Adjusted probabilities of 1-year survival were 73% (95% CI, 60% to 84%) in the cryopreserved graft group and 91% (95% CI, 86% to 94%) in the noncryopreserved graft group. These data support the use of noncryopreserved grafts whenever possible in patients with severe aplastic anemia.

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Section: Discussionmentioning

confidence: 73%

Section: Discussionmentioning

confidence: 78%

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

See 2 more Smart Citations

Hematopoietic Cell Transplantation with Cryopreserved Grafts for Severe Aplastic Anemia

Eapen¹,

Zhang²,

Tang³

et al. 2020

Biology of Blood and Marrow Transplantation

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“…Prior to SCT, hemoglobin was 78 g/L, platelets 32 × 10 9 /L, and ferritin level 4180 μg/L. Conditioning consisted of rabbit ATG (Sanofi) 2 mg/kg on days (D) −9 and −8, followed by fludarabine 30 mg/m 2 and cyclophosphamide 30 mg/kg from D‐5 to D‐2 with total body irradiation 2 Gy on D‐1 7 . GVHD prophylaxis consisted of mycophenolate mofetil 1 g TID from D + 1 to D + 50 and tacrolimus from D‐3 to D + 180 (target levels: 8‐12 nmol/L) tapered completely 1‐year post‐SCT.…”

Section: Case Presentationmentioning

confidence: 99%

Single UM171‐expanded cord blood transplant can cure severe idiopathic aplastic anemia in absence of suitable donors

Claveau

Cohen

Ahmad

et al. 2020

European J of Haematology

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Allogeneic stem cell transplantation (SCT) remains the best treatment for severe aplastic anemia. The current priority order for selection of a stem cell donor is an HLA-identical sibling, a matched unrelated donor, and a haplo-identical donor when the first two are unavailable. 1 Cord blood (CB) transplant also offers a curative option, although few adult cases have been reported due to insufficient number of progenitor cells leading to high risk of graft failure and infections. 1,2 The use of two CB units may accelerate engraftment but has fallen into disfavor due to higher costs of procurement and significant incidence of acute graft-vs-host disease (GVHD). 3 CB expansion can fasten hematologic reconstitution, but no experience in severe aplastic anemia has yet been reported, likely due to fear of graft rejection and lack of availability of expanded CBs. 3 UM171, a first-in-class pyrimidoindole derivative, allows expansion of hematopoietic stem cells by an average of 35-fold within 7 days by enhancing the human long-term-repopulating hematopoietic SC self-renewal independently of aryl hydrocarbon receptor suppression. 4 With a median follow-up of 18 months, a recent phase I/II study in 22 patients with high-risk malignancies has shown that UM171-expanded CB grafts can lead to prompt engraftment, a

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Treatment of newly diagnosed severe aplastic anemia in children: Evidence‐based recommendations

Shimano,

Rothman,

Allen

et al. 2024

Pediatric Blood & Cancer

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Severe aplastic anemia (SAA) is a rare potentially fatal hematologic disorder. Although overall outcomes with treatment are excellent, there are variations in management approach, including differences in treatment between adult and pediatric patients. Certain aspects of treatment are under active investigation in clinical trials. Because of the rarity of the disease, some pediatric hematologists may have relatively limited experience with the complex management of SAA. The following recommendations reflect an up‐to‐date evidence‐based approach to the treatment of children with newly diagnosed SAA.

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Choice of conditioning regimens for bone marrow transplantation in severe aplastic anemia

Abstract: Key Points Flu/Cy/ATG and Cy/ATG regimens offer the best survival for matched-sibling BMT. Transplantation in patients aged ≥30 years is associated with higher mortality after matched-sibling and unrelated donor BMT.

Cited by 47 publications

References 28 publications

Hematopoietic Cell Transplantation with Cryopreserved Grafts for Severe Aplastic Anemia

Hematopoietic Cell Transplantation with Cryopreserved Grafts for Severe Aplastic Anemia

Single UM171‐expanded cord blood transplant can cure severe idiopathic aplastic anemia in absence of suitable donors

Treatment of newly diagnosed severe aplastic anemia in children: Evidence‐based recommendations

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