Bone marrow transplantation in chronic granulomatous disease

Kamani, Naynesh; August, Charles S.; Douglas, Steven D.; Burkey, Edith; Etzioni, Amos; Lischner, Harold W.

doi:10.1016/s0022-3476(84)80354-6

Cited by 35 publications

(9 citation statements)

References 18 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…As a therapeutic alternative to conventional treatment, HSCT is curative [10], especially in patients with no inflammatory or infectious lesions at transplant. However, HSCT carries a high risk of complications and death.…”

Section: Discussionmentioning

confidence: 99%

Clinical features and prognoses of 23 patients with chronic granulomatous disease followed for 21 years by a single hospital in Japan

et al. 2008

View full text Add to dashboard Cite

In this paper, we examined the details of severe infections, treatment efficacies, and the prognoses of 23 Japanese patients with chronic granulomatous disease (CGD). We described the mean ages at diagnosis and follow-up, which were 2.8 years (range, 0.7-10 years) and 14.9 years (range, 0.2-28.4 years), respectively. There were three deaths, two from Aspergillus pneumonia and one from liver abscess. Eighteen of the 23 patients (78%) had a complete loss of gp91phox, and three had p22-phox and one had p67phox deficiencies. Aspergillus species were found in 45% of 174 severe infections. The mean height and weight of the 20 surviving patients were -0.8 +/- 1.3SD and -1.9 +/- 1.9SD below the means for age, respectively. Short stature and underweight (below the 10th percentile of the means) for age were seen in 22% and 17% of the patients, respectively. This growth retardation reflects the severity of the disease. At 20 years of age, there was 87% survival. Ongoing prophylaxis with trimethoprim-sulfamethoxazole (TMP-SMX) or antifungal drugs was given in 16 and 11 patients, respectively. Interferon-gamma (IFN-gamma) was given once a week to 14 patients. Four patients underwent hematopoietic stem cell transplantation (HSCT) and are currently well. There were infections observed in three of 21 identified related carriers of X-linked CGD. A carrier with a liver abscess had 5% normal neutrophils during the acute phase of infection, which returned to 40% normal neutrophils after recovery. The high survival rate in this hospital results from regular follow-up and prophylaxis with TMP-SMX and anti-fungal drugs beginning at the time of diagnosis, along with treatment with weekly IFN-gamma.

show abstract

Section: Discussionmentioning

confidence: 99%

Clinical features and prognoses of 23 patients with chronic granulomatous disease followed for 21 years by a single hospital in Japan

et al. 2008

View full text Add to dashboard Cite

show abstract

“…The 4 deaths occurred only in the group of patients with pre-existing fungal infections refractory to all conventional treatments. We can therefore conclude that HLA-genoidentical HSCT, hitherto reported only in individual CGD patients, [6][7][8][9][10][11][17][18][19][20][21][22] is a valid alternative to conventional treatment.…”

Section: Discussionmentioning

confidence: 99%

Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft: a survey of the European experience, 1985-2000

Seger

Güngör

Belohradsky

et al. 2002

Blood

260

185

View full text Add to dashboard Cite

“…[9][10][11][12][13][14][15][16][17][18][19] In these cases, complications occurring after transplant generally correlated with the severity of the basic disease: patients with recurrent bacterial and fungal infections gradually leading to organ disability had a worse outcome than patients transplanted early on in the course of the disease. [9][10][11][12][13][14][15][16][17][18][19] The indication, therefore, is that BMT should preferably be performed early on in the disease, if the procedure-related risks can be reduced.…”

Section: Discussionmentioning

confidence: 99%

“…1,[7][8][9] Allogeneic bone marrow transplantation is a therapeutic option that has been used in CGD patients since 1976 with varying results. [9][10][11][12][13][14][15][16][17][18][19] However, the procedure continues to be controversial for CGD, as well as other genetic diseases, as it still carries a considerable risk to the recipient, and some physicians feel that it should be restricted to selected, very severe, cases in which prophylactic therapy is problematic. 17,20 Generally speaking, BMT results in CGD are better when the patient is transplanted in an infection-free interval, before septic foci and irreversible organ damage have occurred.…”

mentioning

confidence: 99%

Donor lymphocyte infusion post-non-myeloablative allogeneic peripheral blood stem cell transplantation for chronic granulomatous disease

Nagler

Ackerstein

Kapelushnik

et al. 1999

Bone Marrow Transplant

View full text Add to dashboard Cite

Summary:Chronic granulomatous disease (CGD) is a primary immunodeficiency disease symptomized by failure to generate superoxide and recurrent bacterial and fungal infections. Allogeneic bone marrow transplantation (BMT) is one of the therapeutic options available. However, it presents considerable risk to the recipient, especially if the patient is already at an advanced stage of disease, after repeated bacterial and fungal infections and organ damage. We present a case report of a 6-year-old child with long-standing CGD, severe clubbing, and jeopardized pulmonary function after multiple bacterial pulmonary infectious episodes, who had failed treatment with sulphamethazole trimethoprim, multiple antibiotic courses, itraconazole, as well as steroid and interferon-␥ therapy. He underwent allogeneic peripheral blood stem cell transplantation (alloPBSCT) from his HLA-matched MLC non-reactive sister following non-myeloablative conditioning. His ANC did not fall below 0.2 ؋ 10 9 /l, his lowest WBC was 0.6 ؋ 10 9 /l, and his platelets did not fall below 28 ؋ 10 9 /l. He had normal engraftment, with no mucositis or organ toxicity. Neither parenteral nutrition nor platelet infusions were necessary. Partial donor chimerism following alloPBSCT was converted to full donor chimerism and superoxide production reverted to normal after donor lymphocyte infusions (DLI) from his HLA-matched sister. Twenty four months post transplant the patient is well, with stable and durable engraftment, 100% donor chimerism, normal superoxide production, no GVHD, and stabilization of his pulmonary condition. We suggest that alloPBSCT preceded by non-myeloablative conditioning and followed by DLI may constitute a successful

show abstract

Bone marrow transplantation in chronic granulomatous disease

Cited by 35 publications

References 18 publications

Clinical features and prognoses of 23 patients with chronic granulomatous disease followed for 21 years by a single hospital in Japan

Clinical features and prognoses of 23 patients with chronic granulomatous disease followed for 21 years by a single hospital in Japan

Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft: a survey of the European experience, 1985-2000

Donor lymphocyte infusion post-non-myeloablative allogeneic peripheral blood stem cell transplantation for chronic granulomatous disease

Contact Info

Product

Resources

About