Successful haplo-identical stem cell transplantation in a patient with chronic granulomatous disease

Ref ID: 18608


M. Hönig (1), T. Niehues (2), S. Knirsch (1), C. Schütz (1),
M. Sparber-Sauer (1), V. Mohr (1), W. Friedrich (1), A. Schulz (1)

Author address:

(1)University Hospital (Ulm, DE); (2)HELIOS Klinikum
(Krefeld, DE)

Full conference title:

Annual Meeting of the EBMT, 37th


Chronic granulomatous disease (CGD) is a primary immunodefi ciency characterized by the defi cient generation of reactive
oxygen metabolites by phagocytic cells. As a decisive prophylaxis and antibiotic therapy for bacterial and fungal infections
usually allows the control of infections for many years, the indication for Hematopoietic Stem Cell Transplantation (HSCT) has
been confi ned to patients for whom HLA-identical donors can
be identifi ed.
We report on a patient who was diagnosed with X-linked
CGD at birth (positive family history). Despite antibiotic (Cotrimoxazole) and antifungal (Itraconazole) prophylaxis since
early infancy he suffered from recurrent lymphadenitis, chonic
infl ammatory bowel disease, multiple bacterial pneumonias
and at the age of 10 years developed pulmonary aspergillosis
complicated by fungal osteomyelitis of his 4th rib. This complication could neither be controlled by repetitive surgery nor
systemic antibiotic and antifungal therapy for a period of more
than 4 months. As no HLA-identical donor could be identifi ed
he received a T cell depleted haploidentical transplant (donor:
father; CD34+:1x10e7/kg; CD3+: 0,8x10e4/kg) after conditioning with Busulfan (i.v. 17,6mg/kg), Fludarabine (150 mg/m2
Thiotepa (10 mg/m2
) and Campath (1 mg/kg). Except for a prolonged period of 12 months with low T cell counts the transplant course and follow up of more than 1 year was completely
Summary: In selected patients with CGD and invasive infections not manageable with conservative or surgical measures,
HLA-haploidentical HSCT is a therapeutic option. As patients
with CGD have an isolated defect of phagocytic cells and normal lymphocyte function they are most probably less threatened by viral disease after T cell depleted HSCT compared to
patients with malignancies or primary T cell defi ciencies.

Abstract Number: P602

Conference Year: 2011

Link to conference website: NULL

New link: NULL

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