Bone-Marrow Transplantation for Immunodeficiencies and Osteopetrosis: European Survey, 1968-1985

Fischer, Alain; Friedrich, Wilhelm; Levinsky, R J; Vossen, Jaak M.; Griscelli, C; Kubanek, B; Morgan, Gareth J.; Wagemaker, Gerard; Landais, Paul

doi:10.1016/s0140-6736(86)90477-0

Cited by 174 publications

(46 citation statements)

References 24 publications

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“…Only a few cases of successful aHSCT have been described previously. [9][10][11][12][13][14] Schmid et al 26 recently reported their experience with aHSCT for 10 patients with type 2 GS. At a medium follow-up of 3.4 years, the OS was 70%.…”

Section: Discussionmentioning

confidence: 99%

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Hematopoietic SCT in children with Griscelli syndrome: a single-center experience

Al-Ahmari

Al-Ghonaium

Al-mansoori

et al. 2010

Bone Marrow Transplant

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In total, 11 consecutive pediatric patients with Griscelli syndrome (GS) type 2, who received allogeneic hematopoietic SCT (aHSCT) at our center between 1993 and 2007, were reviewed. The median age at transplantation was 8.2 months (range, 4-36.3 months) and the median time from diagnosis to transplantation was 3.7 months (range, 1.4-19.5 months). Seven patients developed an accelerated phase and were treated with chemotherapy before transplantation. At the time of transplantation, all patients were in clinical remission. The source of grafts was matched-related marrows in eight patients and partially mismatched unrelated cords in three patients. All patients were engrafted at a median time of 15 days (range, 12-36 days). Grade I-II acute GVHD and venoocclusive disease occurred in three and one patient, respectively. A total of 10 patients are now alive and disease free at a median of 4.8 years post-HSCT. The post transplant course was complicated by CMV infection in four patients. One patient died in remission from septic shock, 6 months after transplantation. Chimerism studies at the last contact are available for nine patients: six patients have complete donor chimerism and three have stable mixed chimerism. Early aHSCT from matchedrelated donors or unrelated cord blood for children with GS is feasible.

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Section: Discussionmentioning

confidence: 99%

“…To date, few cases of aHSCT in patients with GS have been reported. [9][10][11][12][13][14] Materials and methods Table 1. The median age at diagnosis was 6.8 (range, 0-17.2) months.…”

Section: Introductionmentioning

confidence: 99%

Hematopoietic SCT in children with Griscelli syndrome: a single-center experience

Al-Ahmari

Al-Ghonaium

Al-mansoori

et al. 2010

Bone Marrow Transplant

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“…The problematic engraftment of B cells is typical of SCID transplants as only 50% of these patients are chimeric for donor B lymphocytes (however they often achieve reconstitution of B cell function). 18 In our patient there has been a gradual increase in the B cell percentage which was 0-1% for several months post transplant and is now 8%. The B cell function seems to be within the normal range as evidenced by the PWM stimulation test and by the normal specific antibodies response to immunization.…”

Section: Discussionmentioning

confidence: 46%

Successful human umbilical cord blood stem cell transplantation without conditioning in severe combined immune deficiency

Toren

Nagler

Amariglio

et al. 1999

Bone Marrow Transplant

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Summary:A 2-month-old girl with severe combined immunodeficiency (SCID), presented with mild staphylococcal skin infection, lymphopenia, low T cell number, absence of B cells, high number of NK cells, and a negligible response to mitogens. Since her older brother died as a result of SCID 2 years earlier, cord blood was harvested from a sister born 2. years earlier, who was normal and fully matched both by serology and molecular typing. In view of her clinical condition and in spite of a high number of NK cells with normal activity, HUCBT without preparative conditioning was performed. No G-CSF was administered. Engraftment with mixed chimerism was evident 3 weeks post transplantation. There were no peritransplantation complications. Eighteen months post transplantation, the girl is in excellent condition, blood counts are normal, T cell engraftment is complete, B cell engraftment is proceeding gradually, and the mitogen stimulation tests are normal. Due to the unique nature of HUCB hematopoietic cells, engraftment without conditioning may be possible in patients with SCID with fully matched donors. This is the first HUCBT performed without conditioning. Keywords: severe combined immunodeficiency (SCID); human umbilical cord blood transplantation (HUCBT); microsatellite sequences; mixed chimerism Severe combined immunodeficiency (SCID) is a heterogenous, lethal, congenital disorder of lymphoid progenitors resulting in the failure of T cells to respond to mitogens and specific antigens, and the inability of B cells to produce specific antibodies. SCID immunophenotypes can be classified according to the presence (B+ SCID) or absence (BϪ SCID) of B cells in the peripheral blood of the patients. 1 There are several causes for the more common B+ SCID

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“…6,15 The success rate for T cell-depleted HLA-mismatched transplants in severe combined immunodeficiencies is not significantly poorer than for HLA-matched transplants (57 vs 68%). 16 Patients with hemophagocytic lymphohistiocytosis who have received HLA-haploidentical transplants with T cell depletion face a 30% risk of failed engraftment. 17 To overcome this problem we gave large numbers of stem cells to our patient (15.4 × 10 6 CD34-positive cells/kg recipient weight).…”

Section: Discussionmentioning

confidence: 99%

Griscelli syndrome: report of the first peripheral blood stem cell transplant and the role of mutations in the RAB27A gene as an indication for BMT

Schuster

Stachel

Schmid

et al. 2001

Bone Marrow Transplant

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Bone-Marrow Transplantation for Immunodeficiencies and Osteopetrosis: European Survey, 1968-1985

Cited by 174 publications

References 24 publications

Hematopoietic SCT in children with Griscelli syndrome: a single-center experience

Hematopoietic SCT in children with Griscelli syndrome: a single-center experience

Successful human umbilical cord blood stem cell transplantation without conditioning in severe combined immune deficiency

Griscelli syndrome: report of the first peripheral blood stem cell transplant and the role of mutations in the RAB27A gene as an indication for BMT

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