Thalassemia-free and graft-versus-host-free survival: outcomes of hematopoietic stem cell transplantation for thalassemia major, Turkish experience

Yeşilipek, Akif; Uygun, Vedat; Küpesiz, Alphan; Karasu, Gülsün; Öztürk, Gülyüz; Ertem, Mehmet; Şaşmaz, İlgen; Daloğlu, Hayriye; Güler, Elif; Hazar, Volkan; Fışgın, Tunç; Sezgin, Gülay; Kansoy, Savaş; Kuşkonmaz, Bülent Barış; Akıncı, Burcu; Özbek, Namık; İnce, Elif; Öztürkmen, Seda; Küpesiz, Funda Tayfun; Yalçın, Koray; Anak, Sema; Bozkurt, Ceyhun; Karakükçü, Musa; Küpeli, Serhan; Albayrak, Davut; Öniz, Haldun; Aksoylar, Serap; Okur, Fatma Visal; Albayrak, Canan; Yenigürbüz, Fatma Demir; Bozkaya, İkbal Ok; İleri, Talia; Gürsel, Orhan; Karagün, Barbaros Şahin; Kintrup, Gülen Tüysüz; Çelen, Safiye Suna; Elli, Murat; Aksoy, Başak Adaklı; Yılmaz, Ebru; Tanyeli, Atila; Akyol, Şule Turan; Siviş, Zuhal Önder; Özek, Gülcihan; Uçkan, Duygu; Kartal, İbrahim; Atay, Didem; Akyay, Arzu; Bilir, Özlem; Çakmaklı, Hasan Fatih; Kürekçi, Emin; Malbora, Barış; Akbayram, Sinan; Demír, Ahmet; Kılıç, Suar Çakı; Güneş, Adalet Meral; Zengin, Emine; Gözmen, Salih; Antmen, Ali Bülent

doi:10.1038/s41409-022-01613-w

Cited by 11 publications

(8 citation statements)

References 34 publications

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“…Thalassaemia is a public health problem with its high incidence and huge socioeconomic burden. HSCT, a cure for severe thalassaemia, has been used worldwide 4,5,10,18,24–26 . HIDs are ideal because of their accessibility and willingness to donate.…”

Section: Discussionmentioning

confidence: 99%

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Haploidentical transplant for paediatric patients with severe thalassaemia using post‐transplant cyclophosphamide and methotrexate: A prospectively registered multicentre trial from the Bone Marrow Failure Working Group of Hunan Province, China

Gong

Chen

et al. 2022

Br J Haematol

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Haploidentical transplantation strategies for patients with transfusion-dependent thalassaemia (TD-TM) remain to be investigated. In this study, 54 paediatric patients with TD-TM were treated with a novel approach using post-transplant cyclophosphamide (PTCy) and low-dose methotrexate (LD-MTX), following a myeloablative regimen. The incidence of neutrophil and platelet engraftment was 96.3% ± 2.6% and 94.4% ± 3.1% respectively. The cumulative incidence of grades II-III acute graftversus-host disease (GVHD) was 13.8% ± 4.8% at 100 days. At three years, the cumulative incidence of chronic GVHD was 28.5% ± 8.5%. With a median follow-up of 520 days (132-1325 days), the overall survival (OS) and event-free survival (EFS) were 98.1% ± 1.8% and 90.7% ± 3.9% respectively. Compared with the low-dose cyclophosphamide (CTX) conditioning regimen (120 mg/kg), the high-CTX regimen (200 mg/ kg) achieved a higher incidence of stable engraftment (100% vs 66.7% ± 15.7%, p = 0.003), a comparable incidence of grades II-III acute GVHD, a lower incidence of chronic GVHD (20.2% ± 8.3% vs 66.6% ± 19.2%, p = 0.011), and better overall survival (100% vs 88.9% ± 10.5%, p = 0.025) as well as EFS (95.6% ± 3.1% vs 66.7% ± 15.7%, p = 0.008). Our results using unmanipulated haploidentical grafts and PTCy with LD-MTX in TD-TM are encouraging. (chictr.org.cn ChiCTR1800017969)How to cite this article: Hu J, Gong S, Chen K, Yang R, Wang L, Yang K, et al. Haploidentical transplant for paediatric patients with severe thalassaemia using post-transplant cyclophosphamide and methotrexate: A prospectively registered multicentre trial from the Bone Marrow Failure Working Group of Hunan

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Section: Discussionmentioning

confidence: 99%

“…HSCT, a cure for severe thalassaemia, has been used worldwide. 4,5,10,18,[24][25][26] HIDs are ideal because of their accessibility and willingness to donate. However, transplantation from HIDs for TD-TM was limited and was considered experimental.…”

Section: Discussionmentioning

confidence: 99%

Haploidentical transplant for paediatric patients with severe thalassaemia using post‐transplant cyclophosphamide and methotrexate: A prospectively registered multicentre trial from the Bone Marrow Failure Working Group of Hunan Province, China

Gong

Chen

et al. 2022

Br J Haematol

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“…The diagnosis and clinical grading of aGVHD were based on the criteria established by the 1994 Consensus Conference on Acute GVHD. 26…”

Section: Definitionsmentioning

confidence: 99%

Effect of serum panel reactive antibodies on allogeneic hematopoietic stem cell transplantation in pediatric thalassemia patients: A single‐center experience

Malbora,

Sarbay,

Dogusan

et al. 2023

Pediatric Transplantation

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BackgroundThe aim of this study was to assess the impact of serum panel reactive antibodies (PRA) on the outcomes of allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric thalassemia patients.MethodsA total of 73 pediatric patients with thalassemia were included in this single‐center study. Pre‐transplant PRA levels were evaluated, and the patients were divided into two groups: PRA‐negative (group 1; n = 44) and PRA‐positive (group 2; n = 29). Patient characteristics, including age, gender, donor type, stem cell source, and HLA compatibility, were analyzed. Transplant outcomes, including engraftment, transfusion requirements, and transplant‐related complications, were compared between the two groups. Further subgroup analysis was performed based on MFI values.ResultsAt the time of transplantation, patients in group 1 were younger than those in group 2 (p = .008). The number of fully matched donors within the family (MSD and MFD) was significantly higher in group 1 (p = .049). Additionally, Rh blood group incompatibility was higher in group 2 (p = .03). There was no statistically significant difference in the engraftment days of neutrophils, platelets, and erythrocytes between the two groups. The frequency of poor graft function and graft failure was higher in the group 2, but there was no statistically significant difference. Post‐transplant transfusion requirements for platelets and red blood cells were significantly higher in the group 2 (p < .001). Transplant‐related complications such as VOD, PRES, and aGvHD were more common in the group 2, but no statistical significance was detected.ConclusionsSerum PRA in pediatric thalassemia patients may impact the outcomes of HSCT. PRA‐positive patients had higher rates of blood product transfusion requirements. Although poor graft function, graft failure, and post‐transplant complications were more common in the group 2, statistical significance was not observed. Identifying patients with high PRA levels can assist in optimizing transplant strategies and post‐transplant care, leading to improved outcomes for the patients.

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“…In Turkey, 87.5% of the 6620 stem cell transplants performed in childhood are allogeneic HSCTs, and 53.5% of these transplants were performed on non-malignant diseases, mostly on thalassemia major patients. In the malignant disease group, ALL is the most common followed by AML and other diseases (5).…”

Section: Indications For Hsct In Childrenmentioning

confidence: 99%

Hematopoetic stem cell transplantation in children

Albayrak¹

2022

J Exp Clin Med

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Bone marrow transplantation is now called hematopoietic stem cell transplantation (HSCT) because, apart from bone marrow, peripheral blood and umbilical cord blood can also be used as stem cell sources. HSCT can be performed in three ways. One is autologous stem cell transplantation. The other is allogeneic stem cell transplantation. Another type of transplantation applied in recent years is haploidentic transplantation. More than 20% of all allogeneic HSCTs are performed in patients under 20 years of age. HSCT has become a well-established lifesaving treatment procedure for many patients with hematological malignancies, hemoglobinopathies, inborn errors, or bone marrow failure syndromes. HSCT is now integrated as an essential part in many treatment concepts and protocols. HSCT is a treatment that provides an increase on success compared to other treatments when it is performed in suitable patients and in accordance with the standards. Selection of the appropriate transplant type, appropriate donor, appropriate preparation regimen, and close monitoring of complications are essential.

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Thalassemia-free and graft-versus-host-free survival: outcomes of hematopoietic stem cell transplantation for thalassemia major, Turkish experience

Cited by 11 publications

References 34 publications

Haploidentical transplant for paediatric patients with severe thalassaemia using post‐transplant cyclophosphamide and methotrexate: A prospectively registered multicentre trial from the Bone Marrow Failure Working Group of Hunan Province, China

Haploidentical transplant for paediatric patients with severe thalassaemia using post‐transplant cyclophosphamide and methotrexate: A prospectively registered multicentre trial from the Bone Marrow Failure Working Group of Hunan Province, China

Effect of serum panel reactive antibodies on allogeneic hematopoietic stem cell transplantation in pediatric thalassemia patients: A single‐center experience

Hematopoetic stem cell transplantation in children

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