Allogeneic Hematopoietic Stem Cell Transplantation for Inherited Disorders: Experience in a Single Center

Ringdén, Olle; Remberger, Mats; Svahn, Britt-Marie; Barkholt, Lisbeth; Mattsson, Jonas; Aschan, J; Blanc, Katarina Le; Gustafsson, Britt; Hassan, Zuzana; Omazic, Brigitta; Svenberg, Petter; Solders, Göran; Döbeln, Ulrika von; Winiarski, Jacek; Ljungman, Per; Malm, Gunilla

doi:10.1097/01.tp.0000181457.43146.36

Cited by 64 publications

(51 citation statements)

References 40 publications

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“…The fourth patient was given fludarabine 30 mg/m2 for 6 days and treosulfan 12 mg/kg for 3 days. 15 Patients A-C were treated with thymoglobulin 8 mg/kg (Genzyme, Cambridge, MA, USA) and patient D was given alemzumab (Genzyme). Immunosuppression was cyclosporine and four doses of MTX.…”

Section: Transplantationmentioning

confidence: 99%

Hematopoietic SCT: a useful treatment for late metachromatic leukodystrophy

Solders

Martin

Andersson

et al. 2014

Bone Marrow Transplant

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In metachromatic leukodystrophy (MLD), the deficiency of the lysosomal enzyme arylsulfatase A (ARSA) leads to demyelination in the central and peripheral nervous system and ultimately to death. Allogeneic hematopoietic SCT (HSCT) is currently the only treatment for adult and late-onset juvenile MLD, although it is still in question because of insufficient follow-up. We wanted to determine whether HSCT could halt the progression of adult and late-onset juvenile MLD. Four treated unrelated patients and three untreated siblings were included in the study, and followed regularly for up to 18 years after transplantation. The patients were assessed from clinical examination, ARSA enzyme levels, magnetic resonance imaging of the brain and neuropsychological and neurophysiological tests. In the treated patients, ARSA levels were normal up to 18 years after transplantation. The parameters evaluated stabilized and remained stable after a latency period of 12-24 months. Two patients live normal lives, partially in a protected environment. The other two patients stabilized at a low cognitive and functional level. One of the controls is demented, one is in a vegetative state and one died. We conclude that, in comparison with their untreated siblings, HSCT halted the progression of the disease in our treated patients.

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Section: Transplantationmentioning

confidence: 99%

Hematopoietic SCT: a useful treatment for late metachromatic leukodystrophy

Solders

Martin

Andersson

et al. 2014

Bone Marrow Transplant

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“…Five of these patients had graft rejection (Table 3). DLI was administered at a median time of 3 months (range [1][2][3][4][5][6][7][8][9][10][11][12][13], and the median number of infusions was one (range 1-5). Median cell dose was 1 Â 10 6 (range 1 Â 10 5 -1 Â 10 8 ) T cells/kg recipient.…”

Section: Management Of Patients With High MCmentioning

confidence: 99%

“…1,2 The aim of transplantation in these diseases is to achieve sustained engraftment to improve hematopoietic function, provide immune competence or increase or normalize enzyme deficiency. Chimerism is commonly used to monitor engraftment after AHSCT.…”

Section: Introductionmentioning

confidence: 99%

Allogeneic hematopoietic SCT in patients with non-malignant diseases, and importance of chimerism

Svenberg

Mattsson

Ringdén

et al. 2009

Bone Marrow Transplant

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In this retrospective study, we evaluated the chimerism status and outcome in 58 patients (64 transplants) with non-malignant diseases. Reduced intensity conditioning (RIC) was given in half of the transplants. Mixed chimerism (MC) was defined as 41% recipient cells. Two consecutive samples showing 430% recipient cells were defined as high chimerism (high MC). Patients with high MC and the management of these patients were analyzed in greater detail. The overall survival rate was 87%. In total, 23 transplants were donor chimerism (DC) and 41 transplants showed some degree of MC. The incidence of MC was 78 and 50% after RIC and myeloablative conditioning, respectively (P ¼ 0.04). Acute GVHD of grades II-III was more common in patients with DC (39%) than in patients with MC (8%) (P ¼ 0.002). Owing to high MC, donor lymphocyte infusions were given in 17 cases. The level of MC was reduced in seven cases, unchanged in four cases, increased in one case and there was graft rejection in five cases. A second transplant was carried out in six cases with rejections, five are alive and in remission. We conclude that patients with non-malignant diseases, who develop MC after transplant have less acute GVHD. Despite the high incidence of MC, overall survival is promising.

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“…1,2 Once confined mainly to highly advanced centers in the United States and Europe, HSCT is now available in many centers worldwide. The Sultanate of Oman, with a population of 2.3 million (Omani 1.78 million, expatriate 0.56 million), 3 started its program in 1995 as an initiative of the hematology department at SQU Hospital.…”

Section: Introductionmentioning

confidence: 99%