HCT is currently the treatment of choice for children with severe primary immunodeficiencies (PIDs). Frequently, these patients lack an HLA-identical sibling donor, and umbilical cord blood (UCB) transplantation may be an option; however, experience in this field remains scant. Fifteen children with PID (SCID 11, X-linked lymphoproliferative syndrome 2, Omenn's syndrome 1, Wiskott-Aldrich syndrome 1) received a UCB transplant. The donor was unrelated in 14 cases and related in 1. Median age at transplant was 11.6 months (range, 2.9-68.0) and median weight 7 kg (range, 4-21). Thirteen patients were conditioned with busulphan and cyclophosphamide and 2 with fludarabine and melphalan. Nine patients received antithymocyte globulin. Median NC x 10(7)/kg infused was 7.9 (range, 2.9-25.0) and median CD34 x 10(5)/kg 2.9 (range, 1.0-7.9). All patients engrafted. Median days to >0.5 x 10(9)/l neutrophils was 31. Eight patients developed acute graft-versus-host disease (GvHD) grades II-IV and one chronic GvHD. Viral and fungal infections were frequent. Four patients died: three from GvHD grade IV complicated by infection and one from progressive interstitial lung disease. Five-year survival was 0.73+/-0.12. All surviving patients presented complete immunologic reconstitution. No patient is intravenous immunoglobulin (IVIg) replacement therapy-dependent. UCB transplantation is a valid option for children with PID who lack an HLA-identical sibling donor.

Unrelated cord blood transplantation for severe combined immunodeficiency and other primary immunodeficiencies.

Pulmonary hypertension (PH) is an infrequently reported complication after hematopoietic stem cell transplantation, and its etiology and therapeutic strategies, especially in infants, remain unclear. We report a case of severe PH that developed in an infant with acute leukemia following administration of busulfan as a preconditioner for cord blood transplantation; the case was successfully treated with sildenafil and beraprost, which to our knowledge is the first reported successful use of this regimen in PH following transplantation for infantile leukemia. From a review of all previous reports, use of busulfan in infants may raise the risk of developing PH, and unlike definitive pulmonary veno-occlusive disease, PH in this subgroup may be reversible by early detection and treatment.

Successful treatment of pulmonary hypertension with beraprost and sildenafil after cord blood transplantation for infantile leukemia

Monoclonal antibodies have provided new promise for patients with B-cell malignancies. Rituximab is a monoclonal antibody against B-lymphocytes that express CD20; it is used for the treatment of patients with relapsed or refractory B-cell non-Hodgkin lymphoma. Very few data are available regarding the treatment of children with non-Hodgkin lymphoma with rituximab. In this article, we reported three children with primary refractory/relapsed B-cell-non-Hodgkin lymphoma, who were successfully treated with a combination of intensive chemotherapy protocol plus rituximab. In all three of our cases, the patients are still in complete remission. Our aim is to emphasize the importance of use of rituximab in the treatment of childhood B-cell non-Hodgkin lymphoma.

Use of rituximab in three children with relapsed/refractory Burkitt lymphoma

Precursor B-ALL blasts may be positive for CD20 in up to 50% of cases. There are few reports on the use of the anti CD20 monoclonal antibody, rituximab, in children with B-ALL. We report on two adult patients with precursor B-ALL who developed significant hepatic toxicity during induction chemotherapy. Single agent rituximab was given until recovery of liver enzymes was established. Both patients were able to continue further chemotherapy, underwent an Allo BMT and are now 10-12 months after diagnosis in a continuous CR.

Rituximab monotherapy as interim therapy in precursor B-ALL adults during periods of hepatic toxicity: report of two cases.

The authors describe a 6-year-old boy diagnosed with mediastinal Burkitt lymphoma with tumor invasion into bone marrow and both kidneys. After receiving chemotherapy according to NHL BFM-95 protocol for the high-risk disseminated lymphoma, the patient reached complete remission. He relapsed in the mediastinum at 5 months from the diagnosis. He underwent thoracotomy and tumor mass was removed by inferior lobectomy of right lung. Residual tumor progressed rapidly. Autologous stem cell transplantation could not be performed because of unresponsiveness to cytoreductive chemotherapy. Twenty-three days after the last chemotherapy course, he received rituximab at a dose of 375 mg/m 2 by intravenous infusion weekly, for a total of 8 dose. However, multiple intra-abdominal metastatic lesions were detected at the end of the therapy. Palliative radiotherapy was applied to these sites. He died because of disease progression, 11 months after the diagnosis.

Refractoriness to rituximab monotherapy in a child with relapsed/refractory Burkitt non-Hodgkin lymphoma.

Pulmonary hypertension (PH) is an uncommon, potentially life-threatening disorder of the lung vasculature. The aetiology of PH is not fully clarified; however, it has been associated with various conditions such as heart disease, infection, collagen vascular disease, veno-occlusive disease (VOD), drugs and toxins (Widlitz & Barst, 2003). PH has very rarely been associated with haematopoietic stem cell transplantation (HSCT) and was mostly associated with vasculopathy or VOD (Hackman et al, 1989; Bruckmann et al, 1991; Seguchi et al, 2000). Recently, Steward et al (2003) described patients with malignant infantile osteopetrosis who developed PH posttransplantation. To illustrate that not only osteopetrosis patients are susceptible to PH in the post-HSCT period, we report two cases with severe immunodeficiency syndrome (SCID) that developed this complication.

Pulmonary hypertension in two severe combined immunodeficiency disease patients posthaematopoietic stem cell transplantation

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Pulmonary hypertension in two severe combined immunodeficiency disease patients posthaematopoietic stem cell transplantation