Hydroxyurea treatment for sickle cell disease: impact on haematopoietic stem cell transplantation's outcome

Brachet, Cécile; Azzi, Nadira; Demulder, Anne; Devalck, Christine; Gourdin, Audrey; Gulbis, Béatrice; Klein, Axel; Lê, Phu Quoc; Loop, Michèle; Sariban, Eric; Ferster, Alina

doi:10.1038/sj.bmt.1704443

Cited by 31 publications

(29 citation statements)

References 22 publications

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“…A recent update of a Belgian study indeed argues in favour of this hypothesis. 20 Compliance with life-long treatment is a perceived problem in adults with SCD. 21 However, in the backdrop of a cure, compliance with such treatment which is of paramount importance after an allo-HSCT might be much better, as evidenced in our study.…”

Section: Discussionmentioning

confidence: 99%

A survey on patient perception of reduced-intensity transplantation in adults with sickle cell disease

Chakrabarti

Bareford

2007

Bone Marrow Transplant

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The development of reduced-intensity conditioning (RIC) and the success of BMT for paediatric sickle cell disease (SCD) have raised the possibility of revisiting this prospect in adults as well. In a chronic debilitating disorder managed with supportive therapy, the patients' perception is critical in the advancement of any potential curative therapy. To explore this aspect, we undertook a questionnaire-based survey on 30 adults with SCD. Sixty two per cent of the patients were ready to accept a transplant-related mortality (TRM) 410%; 30% of them a TRM 430%. A risk of graft failure (GF) 410% was acceptable to 64%, with a risk 430% acceptable to 41%. Infertility was acceptable to only 50%. Chronic graftversus-host disease (GVHD) was unacceptable to the majority (80%). Seventy six per cent% of patients had a full sibling and 60% were willing to participate in a clinical trial of RIC transplantation. This survey suggests that the majority of adults with SCD might be willing to consider a curative option such as RIC transplantation even with a high TRM or GF. The major concerns relate to chronic GVHD and infertility. There is an urgent need to explore RIC transplants in SCD patients within the framework of a clinical trial, considering patient perception regarding cure and complications.

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Section: Discussionmentioning

confidence: 99%

A survey on patient perception of reduced-intensity transplantation in adults with sickle cell disease

Chakrabarti

Bareford

2007

Bone Marrow Transplant

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“…20 The more recent approach that the Pesaro group has taken using hydroxyurea and azathioprine appears to be promising and the report of improvement in outcome of sickle cell anemia patients who were on hydroxyurea prior to transplant supports this concept for conditioning. 21,22 There are no randomized trials on different conditioning approaches for BMT in thalassemia published in the English language literature. The basic conditioning regimen that we decided to use was Bu at 16 mg/kg: we did not want to reduce the Bu dose to 14 mg/kg since these were multiply transfused children with an increased risk of rejection, and we kept the dose of Cy at 200 mg/kg and added ALG.…”

Section: Randomized Trial Of Busulfan In Thalassemia M Chandy Et Almentioning

confidence: 99%

Randomized trial of two different conditioning regimens for bone marrow transplantation in thalassemia – the role of busulfan pharmacokinetics in determining outcome

Chandy

Balasubramanian

Ramachandran

et al. 2005

Bone Marrow Transplant

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“…GVHH proflaksisi için ise CsA ve MTX kullanılmaktadır 8,9 . HKHN sonrası kalıcı engraftment sağlanan olguların takibinde, hastalığa ait klinik bulguların gözlenmediği, dalak fonksiyonlarında ve SSS hastalığının düzelmekte olduğu, MRI bulguları ile transplantasyon öncesine göre serebrovasküler kan akımında belirgin artma olduğunun tespit edildiği ve kemik bozukluklarının ilerlemesinin önlenebildiği gösterilmiştir [10][11][12] . Ayrıca birçok hastada pulmoner hasarın ilerlemesinin durdurulduğu ve lineer büyümenin normale döndüğü gözlenmiştir.…”

Section: Hematopoetik Kök Hücre Nakli (Hkhn)unclassified

Orak Hücre Anemisinde Hematopoetik Kök Hücre Transplantasyonu

Tanyeli¹

2014

Arşiv Kaynak Tarama Dergisi

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Sickle cell anemia is one of the most common hemoglobinopathies in the worldwide. Sickle cell anemia characterized by crises and organ failure develops over time. Myeloablative stem cell transplantation is curative but it has been performed in children younger than 16 years of age. Nonmyeloablative protocols are crucial for the future of Hematopoietic Stem-Cell Transplantation for older sickle cell anemia patients with organ failure. A protocol for nonmyeloablative allogeneic hematopoietic stem-cell transplantation that includes total-body irradiation and treatment with alemtuzumab and sirolimus can achieve stable, mixed donor-recipient chimerism. Stem cell transplantation is recommended in the presence of HLA-matched siblings in patients at risk.

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Hydroxyurea treatment for sickle cell disease: impact on haematopoietic stem cell transplantation's outcome

Cited by 31 publications

References 22 publications

A survey on patient perception of reduced-intensity transplantation in adults with sickle cell disease

A survey on patient perception of reduced-intensity transplantation in adults with sickle cell disease

Randomized trial of two different conditioning regimens for bone marrow transplantation in thalassemia – the role of busulfan pharmacokinetics in determining outcome

Orak Hücre Anemisinde Hematopoetik Kök Hücre Transplantasyonu

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