Cases reported "Chediak-Higashi Syndrome"

Filter by keywords:



Filtering documents. Please wait...

1/6. Failure of sustained engraftment after non-myeloablative conditioning with low-dose TBI and T cell-reduced allogeneic peripheral stem cell transplantation.

    We investigated whether a T cell-reduced allogeneic stem cell transplant (SCT) with minimal conditioning and subsequent donor lymphocyte infusions (DLI) could reduce the incidence and severity of GVHD while retaining stable engraftment. Five patients with hematological malignancies (three MM, one CLL, one chediak-higashi syndrome) were conditioned with TBI (200 cGy). One patient additionally received fludarabine (120 mg/m(2)). CsA and mofetyl-mycophenolate (MMF) were administered to prevent GVHD. All patients were grafted with >3 x 10(6)/kg highly purified CD34( ) cells together with 2 x 10(6)/kg CD3( ) cells (three patients) or 1 x 10(5)/kg CD3( ) cells (two patients). Quick hematopoietic recovery and initial mixed donor chimerism was observed. Treatment-related toxicity was minimal in all but one patient who died of treatment-refractory GVHD on day 112. The four other patients only achieved partial donor T cell chimerism. BM and PBMC donor chimerism was lost between day 40 and 209 despite DLI. Three patients are alive with disease and one is in CR. We conclude that T cell-reduced SCT using 200 cGy as the conditioning regimen does not result in stable hematopoietic engraftment. Predominant donor T cell chimerism is not a prerequisite for initial allogeneic hematopoietic proliferation. However for sustained long-term engraftment it is of major importance.
- - - - - - - - - -
ranking = 1
keywords = chimerism
(Clic here for more details about this article)

2/6. chediak-higashi syndrome: hematopoietic chimerism corrects genetic defect.

    chediak-higashi syndrome is a rare autosomal recessive disorder, primarily affecting neutrophils, and is often lethal by the third decade of life. bone marrow transplantation is the only curative therapy currently available. This case describes a child undergoing a bone marrow transplant from a matched sibling donor, resulting in hematopoietic chimerism with only a small percentage of donor neutrophils found long term. The presence of a small percentage of donor neutrophils has resulted in normal development and no increased incidence of infections. Hematopoietic chimerism offers a cure with a potential reduction in the side-effects that result from marrow transplantation and the associated preparative therapies.
- - - - - - - - - -
ranking = 1.5
keywords = chimerism
(Clic here for more details about this article)

3/6. Split chimerism after allogeneic bone marrow transplantation in chediak-higashi syndrome.

    chediak-higashi syndrome (CHS) is a hereditary multiorgan disease associated with a lymphoproliferative disorder termed 'accelerated phase' (AP). As AP is often life-threatening, hematopoietic stem cell transplantation has been proposed as the only curative treatment for CHS. Here, we report a 1-year-old Japanese boy with CHS who received an HLA-matched unrelated BMT at the AP stage, which resulted in split chimerism. We evaluated the chimerism status of isolated leukocytes and found that only a limited population of T and NK cells was of donor origin and the majority of these and other hematopoietic cells was of host origin. Clinical outcome was successful, and the patient is currently alive and well, free of AP and serious infections more than 18 months after BMT.
- - - - - - - - - -
ranking = 1.5
keywords = chimerism
(Clic here for more details about this article)

4/6. Fludarabine and once-daily intravenous busulfan for allogeneic bone marrow transplantation for chediak-higashi syndrome.

    An HLA-identical sibling bone marrow transplant was done for a patient with chediak-higashi syndrome. The preparative regimen included intravenous fludarabine (40 mg/m2/dx4) and busulfan (130 mg/m2/dx4). busulfan was given once daily. Pharmacokinetic studies showed the area under the concentration-time curve of the once-daily intravenous busulfan was similar to that seen with the total daily dose administered with an every-6-hourly regimen. Toxicity was minimal. Myeloid engraftment occurred on day 17 and donor chimerism was complete. Fludarabine and once-daily intravenous busulfan is well tolerated and is adequate for engraftment of sibling transplant in chediak-higashi syndrome.
- - - - - - - - - -
ranking = 0.25
keywords = chimerism
(Clic here for more details about this article)

5/6. Progressive neurologic dysfunctions 20 years after allogeneic bone marrow transplantation for chediak-higashi syndrome.

    Three patients with chediak-higashi syndrome underwent allogeneic bone marrow transplantation between the ages of 2 years 9 months and 7 years. The outcome was uneventful, with sustained mixed chimerism. No subsequent recurrent infections or hemophagocytic syndrome were observed. At the age of 22 to 24 years, these 3 patients developed a neurologic deficit combining difficulty walking, loss of balance, and tremor. Neurologic evaluation demonstrated cerebellar ataxia and signs of peripheral neuropathy. Moderate axon loss and rarefaction of large myelinated fibers were observed on semithin sections of peripheral nerve. Cerebellar atrophy was detected by cerebral magnetic resonance imaging in 2 patients. We also reviewed the very long-term outcome of the other 11 patients with chediak-higashi syndrome who had received bone marrow transplants at our center since 1981. All displayed neurologic deficits or low cognitive abilities.
- - - - - - - - - -
ranking = 0.25
keywords = chimerism
(Clic here for more details about this article)

6/6. Allogeneic bone marrow transplantation in chediak-higashi syndrome.

    A boy with chediak-higashi syndrome (CHS) treated by allogeneic bone marrow transplantation (BMT) is described. He had had several respiratory infections during his first 2 years of life, and at the age of 2.5 years he presented with an accelerated phase of CHS. Despite treatment with ascorbic acid and trimethoprim/sulfamethoxazole, he continued to experience recurrent bacterial infections. Allogeneic BMT was performed with his HLA- and mixed leukocyte culture-identical healthy brother as the donor. The preparative regimen consisted of busulfan and cyclophosphamide, and methotrexate and cyclosporine A were given as prophylaxis for graft-versus-host disease (GVHD). The patient engrafted well, and no symptoms or signs of acute GVHD developed. He then achieved chimerism status, in which half the peripheral blood neutrophils and some of the bone marrow myelopoietic cells displayed Chediak-Higashi granules, and dna analysis showed half the peripheral blood cells to be of donor origin and the other half to be of host origin. The boy is currently alive and well 24 months after transplant. Allogeneic BMT, even with mixed chimerism as a result, is a potentially curative therapy for CHS.
- - - - - - - - - -
ranking = 0.5
keywords = chimerism
(Clic here for more details about this article)


Leave a message about 'Chediak-Higashi Syndrome'


We do not evaluate or guarantee the accuracy of any content in this site. Click here for the full disclaimer.