Successful second bone marrow transplant for Fanconi's anaemia following escalation of conditioning

O’Donnell, Judith L.; Roberts, Ian S.; Fuente, J. De La; Daly, P.; New, Helen; Dokal, Inderjeet

doi:10.1046/j.1365-2141.1997.2493066.x

Cited by 8 publications

(4 citation statements)

References 9 publications

(10 reference statements)

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“…Haematopoietic growth factors (Nemunaitis et al , 1990; Weisdorf et al , 1995), either granulocyte colony‐stimulating factor or granulocyte–macrophage colony stimulating factor, modification of the host immunological status (Ljungman et al , 1996), infusion of autologous frozen stem cells (Mehta et al , 1996; Fouillard et al , 1998) or donor leucocytes (Godder et al , 1998; Remberger et al , 1998), and second allogeneic SCT are the main therapeutic options that can be proposed in case of graft failure. Data on second allogeneic SCTs in this context are scarce (Davies et al , 1994; Miniero et al , 1996; O'Donnell et al , 1997; Grandage et al , 1998), especially in patients with malignant diseases. A recent retrospective study from the Seattle group, including 44 patients with AA who received a second transplant for graft failure between 1970 and 1996, has confirmed that a graft‐versus‐host disease (GVHD) prophylaxis combining cyclosporin A (CsA) and methotrexate was a major predictor of outcome after second transplant (Stucki et al , 1998).…”

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confidence: 99%

Second early allogeneic stem cell transplantations for graft failure in acute leukaemia, chronic myeloid leukaemia and aplastic anaemia

Guardiola

Kuentz²,

Garban

et al. 2000

Br J Haematol

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Summary. In this retrospective multicentre study, we analysed the results of 82 consecutive second early allogeneic transplants for primary (n 28) or secondary (n 54) graft failures performed between 1985 and 1997 in patients with acute leukaemia (n 33), aplastic anaemia (n 29) or chronic myeloid leukaemia (n 20). HLAmatched siblings were used in 64 cases. The same donors were used for both transplants in 56 cases and the first transplant was T-cell depleted in 30 cases. The median age at transplant was 25 years and the median intertransplant time interval was 2 months. Estimates of the 3-year overall survival and day 100 transplant-related mortality were 30% and 53% respectively. A recipient age , 34 years at transplant, an intertransplant time interval $ 80 d and a positive recipient cytomegalovirus serology were predictors of a better outcome. The use of cyclosporin A (CsA) after second transplant had a dramatic impact on outcome, the best results being observed with CsA alone. The day 40 probability of neutrophil recovery was 73%. The use of peripheral blood progenitor cells (PBPCs) was associated with a higher and faster neutrophil recovery. Other factors associated with neutrophil recovery were an intertransplant time interval $ 80 d and a positive recipient cytomegalovirus serology. Therefore, second early allogeneic transplantation for graft failure is an effective treatment, especially if patients can receive CsA for graft-versus-host disease prevention and are retransplanted more than 80 d from first transplant.

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confidence: 99%

Second early allogeneic stem cell transplantations for graft failure in acute leukaemia, chronic myeloid leukaemia and aplastic anaemia

Guardiola

Kuentz²,

Garban

et al. 2000

Br J Haematol

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“…Sustained engraftment has been observed even in the presence of significant HLA allo-antibodies in two (P1-SIB1 and P3-VUD1) out of the four patients, a well-recognised risk factor for graft failure. 22 Moreover, two prominent features in our patients, extensive malformations and the use of androgens before transplant, have previously been associated with a worse outcome. 15 Furthermore, one of the patients (P3-VUD1) had significant iron overload and abnormalities of liver function at the time of SCT.…”

Section: Discussionmentioning

confidence: 99%

Non-TBI stem cell transplantation protocol for Fanconi anaemia using HLA-compatible sibling and unrelated donors

Fuente

Reiss

McCloy

et al. 2003

Bone Marrow Transplant

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Summary:Allogeneic haemopoietic stem cell transplantation (SCT) is the only curative option for severe bone marrow (BM) failure in patients with Fanconi anaemia (FA). We have developed a non total body irradiation (TBI) conditioning protocol consisting of fludarabine (120-150 mg/m 2 ), low dose of cyclophosphamide (40 mg/kg) and antilymphocyte globulin (45 mg/kg). Graft-versus-host disease (GVHD) prophylaxis was with cyclosporin alone for sibling allografts but also included Campath-1 H (days 1-5 post SCT) for the unrelated allografts. We have performed two sibling and two unrelated BM transplants with a follow-up of 11-51 months. All patients experienced minimal toxicity and were discharged from hospital 28-32 days post SCT. Neutrophil and platelet engraftment occurred from days 11 to 19 and 15 to 34, respectively. All patients achieved stable full donor haemopoiesis with normalisation of the peripheral blood count despite one of them having myelodysplasia (MDS) with 8% blasts prior to the SCT. The only site of acute GVHD was in the skin (grade I-II) and only one patient progressed to limited chronic GVHD. This protocol is associated with reduced toxicity and prompt engraftment in FA patients with AA and/or MDS undergoing SCT using sibling or unrelated donors.

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“…5 In this case report, we describe a patient who experienced a second rejection episode after a second allogeneic stem cell transplantation for FA, and who was successfully treated for the second rejection episode by a well-tolerated donor lymphocyte infusion.…”

Section: Discussionmentioning

confidence: 96%

Rejection of the second allogeneic graft in a child with Fanconi anemia reversed by antilymphocyte globulin and donor lymphocyte infusion

Abdelkefi¹,

Othman²,

Ladeb³

et al. 2003

Hematol J

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Rejection after allogeneic bone marrow transplantation for Fanconi anemia (FA) is a complication with a high risk of mortality. We describe a patient who, following a second episode of rejection after a second allogeneic stem cell transplantation, was successfully treated with antilymphocyte globulin, followed by donor lymphocyte infusion. At three and a half years after donor lymphocyte infusion, she is alive with a Karnofsky score of 90%. Her molecular chimerism is of donor origin. Thus, donor lymphocyte infusion can be considered as a therapy option for rejection after allogeneic bone marrow transplantation for FA.

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Successful second bone marrow transplant for Fanconi's anaemia following escalation of conditioning

Cited by 8 publications

References 9 publications

Second early allogeneic stem cell transplantations for graft failure in acute leukaemia, chronic myeloid leukaemia and aplastic anaemia

Second early allogeneic stem cell transplantations for graft failure in acute leukaemia, chronic myeloid leukaemia and aplastic anaemia

Non-TBI stem cell transplantation protocol for Fanconi anaemia using HLA-compatible sibling and unrelated donors

Rejection of the second allogeneic graft in a child with Fanconi anemia reversed by antilymphocyte globulin and donor lymphocyte infusion

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