Allogeneic peripheral blood stem cell transplant in children

Li, Chi Kong; Yuen, Patrick Man Pan; Chik, Ki Wai; Shing, Matthew Ming Kong; Li, Karen; Tsang, Kam Sze; Wong, Ambrose H.; Leung, Ting Fan; Lai, Hsin‐Cheng

doi:10.1002/(sici)1096-911x(199803)30:3<147::aid-mpo3>3.0.co;2-h

Cited by 24 publications

(17 citation statements)

References 17 publications

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“…32,33 Based on this, some investigators 26 have postulated that mobilization with higher doses of G-CSF would result in similar rates of cGVHD compared to those seen after BMT. While the reported incidence of aGVHD with PBPCT is similar compared to BMT, [15][16][17]34,35 several authors have found an increased incidence of cGVHD. Levine et al 36 found a probability of cGVHD of 75% at 1 year after PBPCT in pediatric patients.…”

Section: Discussioncontrasting

confidence: 51%

“…These results were in accordance with those found by others in pediatric patients undergoing PBPCT for malignant and non-malignant diseases. 15,34,35 The probability of cGVHD in this study was higher in the PBPCT group compared to the BMT group although the difference was not statistically significant. This difference is particularly evident after the first year from transplantation.…”

Section: Discussionmentioning

confidence: 99%

“…Because of this, longer follow-up is necessary to address this controversial issue. However, despite differences in cGVHD incidences there is no reported negative influence on event-free survival in pediatric series, 15,34,35,37 which implies a similar TRM and a similar relapse incidence. One could expect a lower probability of leukemia relapse in PBPCT patients because of a cGVHD-related graft-versus-leukemia effect.…”

Section: Discussionmentioning

confidence: 99%

“…Data from these reports suggest that allo-PBPCT appears safe in pediatric patients and donors, and it seems not to be associated with an increase in acute GVHD. [13][14][15][16][17] Taking into account these considerations, we conducted a case-control study in pediatric patients with ALL comparing blood and bone marrow as sources of stem cells for allografting. Endpoints of this study were engraftment kinetics, acute and chronic GVHD incidence, transplant-related mortality (TRM), risk of relapse and event-free survival (EFS).…”

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confidence: 99%

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Matched-pair analysis comparing allogeneic PBPCT and BMT from HLA-identical relatives in childhood acute lymphoblastic leukemia

Vicent

Madero

Ortega³

et al. 2002

Bone Marrow Transplant

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Summary:This multicenter study was designed to evaluate whether allo-PBPCT provides some advantages, if any, over BMT in terms of engraftment kinetics, acute and chronic GVHD incidence, TRM, relapse incidence and survival in acute lymphoblastic leukemia patients (ALL). From January 1995 to December 1999, 67 ALL patients (34 in the PBPCT group and 33 in the BMT group) were included in this study. Median age for both groups was 8 years (range, 1-18). There were 24 patients in first or second CR in the PBPCT group and 28 such patients in the BMT group. Preparatory regimens were TBI-based in 26/34 in the PBPC group and 25/33 in the BMT group. GVHD prophylaxis was CsA alone in 38 patients (18 PBPCT vs 20 BMT) and CsA plus short Mtx in 29 (16 PBPCT vs 13 BMT). Engraftment was achieved in all cases. Median days to neutrophil recovery was 10 (range, 7-18) after PBPCT vs 14 (range, 9-21) after BMT (P Ͻ 0.0001). Platelet engraftment (Ͼ50 ؋ 10 9 /l) was also faster for PBPCT patients (median 13 days, range, 9-40 vs 23 days, range, 15-165) (P Ͻ 0.0001). Acute GVHD grade II-IV incidence was similar in both groups (46.4 ؎ 8.8% vs 42.7 ؎ 8.6%) (P ‫؍‬ 0.45). Probability of chronic GVHD was 50.6 ؎ 12.2% after PBPCT vs 27.8 ؎ 9.2% after BMT (P ‫؍‬ 0.1). Probability of relapse was similar (28.7 ؎ 9.2% for PBPCT vs 27.1 ؎ 8.2% for BMT) (P ‫؍‬ 0.89). There were eight patients who died from transplantrelated complications after PBPCT vs 5 after BMT (P, NS). With a median follow-up of 25 months the eventfree survival probability was 53 ؎ 8.9% for PBPCT vs 54.9 ؎ 9.7% for BMT (P ‫؍‬ 0.54). Using PBPC for allogeneic transplantation in childhood ALL results in faster hematopoietic recovery compared to BM, with a similar incidence of aGVHD, TRM, relapse and disease-

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Section: Discussioncontrasting

confidence: 51%

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

mentioning

confidence: 99%

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Matched-pair analysis comparing allogeneic PBPCT and BMT from HLA-identical relatives in childhood acute lymphoblastic leukemia

Vicent

Madero

Ortega³

et al. 2002

Bone Marrow Transplant

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“…Data from the literature suggest that PSCT appears to be safe for pediatric donors, yields sufficient progenitor stem cells and results in prompt engraftment. 5,[10][11][12][13][14] The median neutrophil engraftment day was 12, and all patients achieved engraftment before day 14 in our series. Mobilization of PBSC and collection by leukapheresis has practical advan- tages for the donor over the traditional marrow harvest, including the avoidance of general anesthesia, blood loss and invasive surgery.…”

Section: Discussionmentioning

confidence: 99%

Peripheral blood stem cell transplantation in children with beta-thalassemia

Yeşilipek

Hazar

Küpesiz

et al. 2001

Bone Marrow Transplant

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Summary:Fifteen patients with beta-thalassemia received an allogeneic peripheral blood stem cell transplant. Median age was 3.5 years (1-15 years). Six were class I, four class II and five class III according to the Pesaro criteria. All of the donors were HLA-phenotypically identical (13 siblings and two parents). Nine patients were given BU ؉ CY and six BU ؉ CY plus ATG as conditioning. All patients received MTX (؉1, ؉3, ؉6) and CsA (9-12 months) post transplant for GVHD prophylaxis. The median neutrophil and platelet engraftment times were day 12 and day 16, respectively. cGVHD was observed in three patients. Two patients died. Thirteen patients are well, and transfusion-independent 2-30 months after PSCT. No recurrences of thalassemia have been seen. Overall and event-free survival were 86.6%. In conclusion, we suggest that PSCT can be considered a safe and effective treatment for children with betathalassemia. Bone Marrow Transplantation (2001) 28, 1037-1040. Keywords: peripheral blood stem cell transplantation; beta-thalassemia; children Stem cell transplantation is considered the only curative treatment approach for the hemoglobinopathies. Successful cure of ␤-thalassemia by bone marrow transplantation (BMT) was first reported by Thomas and associates in 1982. 1 Subsequently, a number of centers have explored this approach as a curative therapy for ␤-thalassemia. The most extensive experience with BMT in ␤-thalassemia was reported by Lucarelli and co-workers 2 in Italy who studied more than 800 transplanted patients. Recently, peripheral blood stem cells (PBSC) have been increasingly used in transplantation. Some authors have suggested that peripheral blood stem cell transplantation (PSCT) has some advantages as compared with bone marrow stem cell transplantation. geneic transplantation appears to be safe for both pediatric donors and patients, leading to rapid hematopoietic engraftment with a similar incidence of acute graft-versushost disease (AGVHD) as is seen after BMT.We report the PSCT experience of our department concerning 15 patients with beta-thalassemia. Materials and methodsFifteen patients with beta-thalassemia received an allogeneic peripheral blood stem cell transplant at Akdeniz University School of Medicine Department of Paediatric Haematology-Oncology. Patient characteristics are shown in Table 1. All of the patients were transfusion-dependent before stem cell transplantation. Fourteen patients had homozygous beta-thalassemia and one had HbS-betathalassemia. Female to male ratio was 9/6 and median age of the patients was 3.5 years (range 1-15 years). Liver biopsy was performed on all patients who were older than 3 years. Six of them were class I, four class II and five were class III according to the Pesaro criteria. 6 All but two patients were HbS antibody-positive. Only one patient (No. 4) was HCV antibody-positive. Thirteen of the donors were fully HLA-matched siblings and two were parents

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