Mv Gazzola

Extracorporeal photochemotherapy may improve outcome in children with acute GVHD

Acute GVHD (aGVHD) is a major cause of morbidity and mortality after unrelated BMT (UBMT). Our purpose was to analyze the role of extracorporeal photochemotherapy (ECP) in controlling grade II–IV aGVHD in children given UBMT. Of 41 consecutive children, 31 developed grade II–IV aGVHD after UBMT: 16 had a good response to steroids (GR group), whereas 15 underwent ECP (ECP group) within 100 days of UBMT. Eligibility criteria for starting ECP were steroid resistance, dependence or viral reactivations. Criteria for judging response to aGVHD treatment were that the resolution of all signs were considered a complete response (CR), at least a 50% improvement was classified as a partial response (PR) and stable or progressive disease was judged as no response (NR). On completing ECP, the CR rate was 73%, whereas the GR group had a CR rate of 56% by day 100. The 2-year overall survival and progression-free survival rates were 57 and 67% in the GR group vs 85 and 87% in the ECP group. Our data seem to suggest that ECP may improve outcome in patients after UBMT. These findings need to be confirmed in a larger population.

Autologous bone marrow transplantation for childhood acute lymphoblastic leukaemia in Italy

From January 1984 to December 1994, ABMT was performed on 154 children (101 males, 53 females; median age 10, range 3–21 years) with ALL and registered for BMT by the AIEOP (Italian Association of Paediatric Haemato-Oncology). All patients were in CR: 98 were in 2nd CR and 56 were in >2nd CR. Fifteen children (9.7%) died of transplant-related mortality. Ninety-five patients (61.6%) relapsed at a median of 5 (range 1–42) months after ABMT. The 8-year EFS according to pre-BMT status was 34.6% (s.e. 4.9) for 2nd CR patients and 10.6% (s.e. 5.6) for patients in >2nd CR. By univariate analysis, site of relapse (isolated extramedullary (IE) vs BM: EFS = 68.5% vs 18.2%; P < 0.0001) and tbi containing regimen (tbi vs no TBI: EFS = 48.1 vs 15.4%; P = 0.0023) were significant factors for 2nd CR patients. When the 2nd CR subset with BM involvement was analysed, TBI became insignificant (EFS = 25.4 vs 11.8%). No factors influenced EFS in patients in >2nd CR. By multivariate analysis, site of relapse was the only significant factor in 2nd CR patients (P < 0.0001). in conclusion, abmt is an effective treatment after one early ie relapse. few patients can be rescued after bm relapse.

Unrelated bone marrow transplantation for high-risk anaplastic large cell lymphoma in pediatric patients : a single center case series

Abstract:  Objectives: The use of allogeneic stem cell transplantation in NHL patients is not yet clearly defined, especially in children and adolescents, but this option offers the advantages of a tumor‐free graft and the possible induction of a graft‐vs.‐tumor effect. Patients and methods: We report the results of four consecutive pediatric patients affected by anaplastic large cell lymphoma (ALCL) and treated with allogeneic stem cell transplantation from an unrelated donor. The conditioning regimen was based on total body irradiation given in association with etoposide in three patients, and with thiotepa and cyclophoshamide in one patient. Graft‐vs.‐host disease (GVHD) prophylaxis consisted of cyclosporin, a short course of methotrexate and rabbit antithymocyte globulin. Results: All patients had rapid engraftment within 3–4 wk for neutrophils and platelets, and achieved a stable full donor chimerism that has been maintained to the last follow‐up visit. One patient later developed a restrictive pneumonopathy. This patient had been heavily pretreated during the course of the disease having suffered four relapses and had received a cumulative dose of bleomycin of 160 mg/m2. After a follow‐up of 11–42 months, all patients are alive in complete hematological and molecular remission; and three of them without any chronic GVHD. Conclusions: The increasing number of volunteer bone marrow donors and the reduced toxicity of unrelated stem cell transplantation, especially in children, make this therapeutic option worth more extensive investigation in the treatment of high‐risk failure ALCL, although more data is needed to evaluate the long‐term benefits. In this regard, the presence of factors predictive of worst outcome such as an early relapse (within 12 months from diagnosis), a refractory or relapsing ALCL and the persistent detection on blood or bone marrow of nucleophosmin‐anaplastic lymphoma kinase protein (NPM‐ALK) transcript may help select the patients eligible to allogeneic related or unrelated stem cell transplantation.

Successful unrelated bone marrow transplantation for Shwachman–Diamond syndrome

A 5-year-old boy with Shwachman–Diamond syndrome underwent unrelated HLA-identical bone marrow transplantation for severe pancytopenia. Conditioning was with busulfan, thiotepa and cyclophosphamide plus rabbit anti-lymphocyte serum. Engraftment for neutrophils and platelets was observed on days +18 and +41, respectively. Transplant-related side-effects were mild and transient. After a follow-up of 32 months, the patient is alive and enjoys a normal life, off any immunosuppressives. Immunological and hematological reconstitution is complete while other phenotypic characteristics (pancreatic insufficiency, short stature, femur dysostosis) are stable. Although experience in this field is scarce, we speculate that bone marrow failure in Shwachman–Diamond syndrome (even if not linked to the appearance of clonal disorders or leukemic transformation) is an indication for bone marrow transplantation and may be associated with a better outcome. Bone Marrow Transplantation (2001) 27, 97–99.

Successful treatment of secondary acute myeloid leukemia relapsing after allogeneic bone marrow transplantation with donor lymphocyte infusion failed to prevent recurrence of primary disease: a case report

We report a case of therapy-related secondary acute myeloid leukemia occurring in a patient during treatment for anaplastic large cell lymphoma. In spite of response to induction chemotherapy and prompt bone marrow transplantation from his matched sister, the patient experienced an early leukemia relapse within 3 months of the transplant. Treatment with oral etoposide for 3 weeks followed by donor lymphocyte infusion achieved a 7-month remission from leukemia without any further treatment. Unfortunately, the patient suffered a recurrence of the primary anaplastic large cell lymphoma that was treated by resuming chemotherapy and local radiotherapy. The patient died 20 months after DLI, still in CR for his leukemia, due to ALCL progression.

Focal nodular hyperplasia of the liver: an emerging complication of hematopoietic SCT in children

Hepatic focal nodular hyperplasia (FNH) is a nonmalignant condition rarely affecting children previously treated for cancer, especially those who received hematopoietic SCT (HSCT). Some aspects of its pathogenesis still remain unclear and a strong association with specific risk factors has not yet been identified. We report here a single institutions case series of 17 patients who underwent HSCT and were diagnosed with FNH, analyzing retrospectively their clinical features and the radiological appearance of their hepatic lesions. We aimed to compare the diagnostic accuracy of ultrasound (US) and magnetic resonance imaging (MRI) and to explore the role of transient elastography (FibroScan) to evaluate the degree of hepatic fibrosis in FNH patients. Our analysis showed an association of FNH with age at transplant ⩽12 years (hazard ratio (HR) 9.10); chronic GVHD (HR 2.99); hormone-replacement therapy (HR 4.02) and abdominal radiotherapy (HR 4.37). MRI proved to be a more accurate diagnostic tool compared with US. Nine out of 12 patients who underwent FibroScan showed hepatic fibrosis. Our study points out that FNH is an emerging complication of HSCT, which requires a lifelong surveillance to follow its course in cancer patients.

TCRαβ CD19 depletion in allogeneic haematopoietic stem cell transplantation performed for Hurler syndrome

TCRαβ CD19 depletion in allogeneic haematopoietic stem cell transplantation performed for Hurler syndrome