Summary:Pure red cell aplasia (PRCA) is a well-known although infrequent hematologic complication after allogeneic bone marrow transplantation. PRCA occurs in cases of major ABO-mismatch between donor and recipient and is believed to be due to inhibition of donor erythroid progenitors by residual host isohemagglutinins. We report a 10-year-old boy with post-hepatitis aplastic anemia (AA) who developed PRCA after HLA-matched familial peripheral blood stem cell transplantation (SCT) following conditioning with Cph 200 mg/kg + ATG 90 mg/kg. Granulocyte engraftment occurred on day +18, platelet engrafted on day +40, while reticulocytopenia at 0% persisted until day +118, and erythroid precursors were totally absent from bone marrow. After a single dose of rituximab 200 mg/m 2 administered on day +118 PRCA resolved and on day +132 the reticulocytes rose to 5.7%. On day +139 the Hb reached 137 g/l and the erythroid lineage in BM increased to 21%. We conclude that due to the rapid recovery from PRCA and lack of side effects, rituximab should be tried as firstline treatment of PRCA after allo-SCT. Bone Marrow Transplantation (2002) 30, 405-407. doi:10.1038/sj.bmt.1703668 Keywords: pure red cell aplasia; allogeneic stem cell transplantation; rituximab Pure red cell aplasia (PRCA) is an infrequent but wellrecognized complication of allogeneic bone marrow transplantation. Most cases of PRCA occur after transplantations with major donor-recipient ABO mismatch and are due to the long persistence of residual titers of natural recipient anti-A, 1 or very rarely, anti-B antibodies, 2 causing inhibition of donor erythroid progenitors. Additional risk factors include use of reduced intensity conditioning and immunosuppression with CsA in the post-transplant
Summary:Allogeneic hematopoietic stem cell transplantation (SCT) from unaffected donors remains the only modality for the correction of hematological abnormalities in Fanconi anemia (FA) patients. We performed four HLA-matched related donor SCT using a novel irradiation and cyclophosphamide-free conditioning regimen. The protocol included fludarabine 150 mg/m 2 , busulfan 4 mg/kg, and antithymocyte globulin 90 mg/kg. Graft-versus-host disease (GVHD) prophylaxis was cyclosporin A, MTX, and daclizumab. The engraftment and occurrence of full stable donor hemopoiesis was rapid in all cases with minimal short-term toxic complications. There were no infections or febrile episodes during the inpatient phase. Three patients developed acute GVHD grade I-II involving gut and skin and one patient progressed to extensive chronic GVHD. The preparative conditioning regimen is safe and associated with low organ toxicity and effective immunosupression for the stable engraftment in FA patients undergoing SCT with matched related donors. Fanconi anemia (FA) is a rare autosomal recessive syndrome of progressive marrow failure, constitutional physical abnormalities of skin, skeleton, kidneys, and cancer susceptibility. 1 FA cells are characterized by chromosomal instability and marked DNA hypersensitivity to crosslinking alkylating agents such as cyclophosphamide or platinum derivatives. 2,3 This hypersensitivity forms the basis for the confirmatory tests for FA with diepoxybutane (DEB), nitrogen-mustard, and others. Bone marrow failure remains the main cause of mortality among the FA patients, followed by complications of stem cell transplantation (SCT) and myeloid and nonmyeloid malignancies. Allogeneic hematopoietic SCT from unaffected sibling donors or suitably matched unrelated donors remains the only curative option for the correction of hematological abnormalities in FA patients. In the past, the use of highdose cyclophosphamide and ionizing radiation in preparative regimens often resulted in excessive organ toxicity and death in the early post transplant period. Low-doses of cyclophosphamide (20-40 mg/kg) combined with a 4-6 Gy of thoraco-abdominal or total body irradiation results in reduced toxicity and substantially improves the outcome for the FA patients transplanted from HLA-matched donors. 4 However, reduced doses of cyclophosphamide may not kill DEB-resistant naturally reverted T-lymphocytes, thereby increasing the risk of rejection in mosaic recipients of unrelated grafts (J Wagner, unpublished). Moreover, longterm follow-up clearly shows that these protocols are still associated with significant regimen-related toxicity and high risk of secondary malignant transformations. 5 More specifically, high incidence and severity of acute and chronic graft-versus-host disease (GVHD) in FA patients, who received cyclosporine alone as the GVHD prophylaxis, significantly contributed to transplantation-related morbidity and mortality, in addition favoring development of late epithelial cancers in irradiated patients. These obs...
Summary:We describe a 5-year-old girl with Ph(؉) CML who received a cord blood transplant in a second accelerated phase after a very early lymphoid blast crisis. She was induced into CR by ALL-directed chemotherapy and then maintained with IFN-␣2b together with weekly rotational chemotherapy. Nineteen months after diagnosis, her mother gave birth to an HLA-compatible sibling, whose cord blood was cryopreserved. The patient's second acceleration occurred 22 months after the CML diagnosis Allogeneic hematopoietic cell transplantation is at present the only potentially curative therapy in patients with CML. 1,2 This type of transplantation is clearly superior to conventional therapy and hence should be considered when treating any child with this disease; however, only a minority of patients have HLA-compatible related donors, and a patient belonging to an ethnic group that is not widely represented in the registries is even less likely than most patients to find a perfectly matched unrelated donor (MUD). 3 Furthermore, marrow delivery can be difficult for centers not accredited by the international registries network. For children experiencing blast crisis who lack a related donor, three goals emerge -to return the disease to its chronic phase, to slow further disease progression and to explore the possibility of performing an allogeneic stem cell transplant. If the first two goals are met, the search for MUD can begin, and if the search is successful, a transplant can be performed. Alternatively, the patient's mother may opt to plan a pregnancy, which, assuming HLA compatibility of the fetus, will allow for the collection of cord blood for transplantation. We report a successful related cord blood transplant in a child with CML who had rapidly progressed to blast crisis. Case reportA 5-year-old girl, the only child of young parents, was admitted in May 1995 with a 2-week history of low-grade fever, leg pain and swelling of the feet. Physical examination revealed inguinal, submaxillar and axillar lymph nodes, enlarged up to 2 cm, moderate hepatomegaly ϩ2 cm and redness, pain and swelling of the right ankle joint. No splenomegaly was present. Her CBC revealed Hb 98 g/l, platelets 533 ϫ 10 9 /l, WBC 72 ϫ 10 9 /l, myeloblasts 3%, promyelocytes 6%, myelocytes 6%, metamyelocytes 10%, bands 9%, segmented 50%, eosinophils 5%, basophils 7%, monocytes 1%, lymphocytes 3%. Bone marrow aspirate showed blast cells 5%, increased to 73% myeloid lineage with myeloblasts 9% and promyelocytes 4%. BM cytogenetics demonstrated Ph chromosome in 100% metaphases. A diagnosis of CML was made and, in the absence of a matched donor, hydroxyurea 50 mg/kg b.w. per day was initiated; however, the patient's clinical state continued to deteriorate, as indicated by her high fever, polyarthritis and splenic enlargement. One month later, 46% blast cells emerged in the peripheral blood which expressed TdT, CD10, CD19, CD22 and HLA DR, whereas T-lineage and myeloid markers were negative. This confirmed a B-lineage blast crisis of CML, and the patien...
Allogeneic stem cell transplantation remains the best option for young patients with SAA. With genetically identical twin as an ideal donor, the majority of SAA patients require appropriate immunosuppression before and after stem cell transplantation to obtain long-term hematopoietic reconstitution. Alkylating agents, used during conditioning, are associated with short- and long-term toxic effects that lead to poor compliance of treatment and could compromise the quality of future life. Three SAA patients, transplanted from genetically identical twins without using alkylating agents during conditioning, showed rapid and sustained hematological reconstitution without any evidence of conditioning-related toxicity.
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