[Successful treatment with prednisolone for air-leak syndrome following interstitial pneumonia after allogeneic hematopoietic stem cell transplantation].

A 13-year-old boy with T lymphoblastic leukemia underwent allogeneic bone marrow transplantation from an HLA-matched sibling in the second remission phase. After the dose of cyclosporine (CyA) was reduced, dyspnea appeared on Day 117. CT revealed diffuse interstitial shadows on the bilateral lungs. The results of broncho-alveolar lavage suggested Pneumocystis jirovecii pneumonia. The dose of trimethoprim-sulfamethoxazole was increased, and steroid therapy was started. The symptoms transiently subsided, but exacerbated with a reduction in the steroid dose. On Day 139, mediastinal and subcutaneous emphysema appeared. We considered that non-infectious interstitial pneumonia was primarily involved in the pathogenesis for the following reasons: the boy was negative for ß-D-glucan early after onset, and there was a correlation between the steroid-dose reduction and condition. The steroid dose was again increased to 80 mg and the symptoms promptly subsided. When late-onset non-infectious pulmonary complications after hematopoietic stem cell transplantation lead to air-leak syndrome, the mortality rate is very high. However, survival may be achieved by intensifying immunosuppressive therapy in the early stage.

Nationwide survey of bisphosphonate therapy for children with reactivated Langerhans cell histiocytosis in Japan.

BACKGROUND: Several studies have suggested that Langerhans cell histiocytosis (LCH) is responsive to treatment with bisphosphonates (BPs). However the efficacy and safety of BPs therapy for childhood LCH is unknown. PROCEDURE: Data on children with LCH who had received BPs therapy were collected retrospectively from hospitals participating in the Japanese Pediatric Leukemia/Lymphoma Study Group. RESULTS: Twenty-one children with histologically proven LCH were identified. Of these, the case histories of 16 children who had been treated with pamidronate (PAM) for disease reactivation were analyzed in detail. The median post-PAM therapy follow-up period was 2.8 years (range: 0.9-9.3 years). The median age at commencement of PAM therapy was 9.4 years (range: 2.3-15.0 years). All children had one or more bone lesions but none had risk organ (RO) involvement. In the majority of the children, six courses of PAM were administered at a dose of 1.0 mg/kg/course at 4-week intervals. In 12 of the 16 children, all active lesions including lesions of the skin (n = 3) and soft tissues (n = 3) resolved. Of these children, eight children had no active disease for a median of 3.3 years post-PAM therapy (range: 1.8-9.3 years). Progression-free survival (PFS) was 56.3 ± 12.4% at 3 years. PFS was significantly higher in children with a first reactivation compared with children experiencing a second or subsequent reactivation. CONCLUSIONS: PAM may be an effective treatment for reactivated LCH with bone lesions. A prospective trial of the efficacy of PAM in recurrent pediatric LCH is warranted.

Outcome of childhood acute lymphoblastic leukemia with induction failure treated by the Japan Association of Childhood Leukemia study (JACLS) ALL F-protocol.

BACKGROUND: Children with acute lymphoblastic leukemia (ALL) who fail to achieve complete remission (CR) after induction therapy (induction failure: IF) have a poor prognosis; however, there have been few prospective studies in patients with IF. PATIENTS AND METHODS: Between April 1997 and March 2005, 27 of 1,237 leukemic patients (2.2%) failed to achieve CR after four- or five-drug induction therapy. Twenty-three of these patients entered the F-protocol study, which mainly consisted of acute-myeloid-leukemia-oriented chemotherapy followed by scheduled hematopoietic cell transplantation (HCT). RESULTS: Seventeen (73.9%) of the 23 patients responded to re-induction chemotherapy with CR. Of note, 15 (93.8%) of 16 patients with Philadelphia-chromosome-negative (non-Ph(+)) ALL achieved CR; in contrast, only 2 (28.6%) of 7 Ph(+) patients achieved CR. Fourteen (82.4%) of 17 patients remained in CR (CCR) until their scheduled HCT, 12 of the 14 with CCR underwent HCT as scheduled, and 6 patients remain in first CR after a median of 78 months (range, 49-107 months). The 5-year overall survival (OS) rates of 16 patients with non-Ph(+) and 7 patients with Ph(+) were 43.8 +/- 12.4% and 14.3 +/- 13.2%, respectively (P = 0.012). The 5-year OS rate of the 17 patients who obtained CR by re-induction therapy and the 6 who did not were 47.1 +/- 12.1% and 0%, respectively (P < 0.001). CONCLUSION: Acute-myeloid-leukemia-oriented chemotherapy followed by scheduled HCT is a promising treatment strategy for non-Ph(+) ALL patients with IF.

Identification of severe combined immunodeficiency by T-cell receptor excision circles quantification using neonatal guthrie cards.

OBJECTIVE: To assess the feasibility of T-cell receptor excision circles (TRECs) quantification for neonatal mass screening of severe combined immunodeficiency (SCID). STUDY DESIGN: Real-time PCR based quantification of TRECs for 471 healthy control patients and 18 patients with SCID with various genetic abnormalities (IL2RG, JAK3, ADA, LIG4, RAG1) were performed, including patients with maternal T-cell engraftment (n = 4) and leaky T cells (n = 3). RESULTS: TRECs were detectable in all normal neonatal Guthrie cards (n = 326) at the levels of 10(4) to 10(5) copies/microg DNA. In contrast, TRECs were extremely low in all neonatal Guthrie cards (n = 15) and peripheral blood (n = 14) from patients with SCID, including those with maternal T-cell engraftment or leaky T cells with hypomorphic RAG1 mutations or LIG4 deficiency. There were no false-positive or negative results in this study. CONCLUSION: TRECs quantification can be used as a neonatal mass screening for patients with SCID.

Effect of transfusion on the venous blood lactate level in very low-birthweight infants.

BACKGROUND: In recent years the blood lactate level can be easily and quickly measured with a small amount of blood, and the availability of an arterial blood lactate level has been reported as an indicator of oxygen deficit in adults. To determine whether venous blood lactate level can serve as such a marker for determining the indications for transfusion, blood lactate and hemoglobin level were monitored before and after transfusion. METHODS: The study subjects consisted of 12 very low-birthweight infants admitted to the neonatal intensive care unit and who had transfusion between June 2005 and June 2007. The data on the blood lactate and hemoglobin were collected retrospectively by referring to the clinical records. RESULTS: A total of 18 transfusions was performed. There was no significant relationship between venous blood lactate and hemoglobin concentration before transfusion. The subjects were classified into two groups according to the lactate level before transfusion: > or =3.3 mmol/L and <3.3 mmol/L. In the high-lactate group the lactate decreased significantly after transfusion (P < 0.01) and it continued to decrease thereafter. In the low-lactate group, however, the lactate remained unchanged. CONCLUSIONS: Venous blood lactate measurements may offer some additional information regarding the optimal time for performing a transfusion. To the authors' knowledge this is the first report to study the changes in lactate levels using venous blood sampling in red blood cell transfusion in very low-birthweight infants.

Decreased platelet aggregation of platelet concentrate during storage recovers in the body after transfusion.

BACKGROUND: Previous reports have shown that storage decrease the ability of PLTs to aggregate in the form of PLT concentrate (PC). Nevertheless, there are few reports that have studied the PLT function in blood samples obtained from recipients after PLT transfusion. In this study, this issue was addressed by examining the ability of PLTs to aggregate after being transfused into the blood stream. STUDY DESIGN AND METHODS: PC was transfused to the patients with extremely low PLT counts resulting from chemotherapy. The maximum extent of PLT aggregation and size of the aggregates were compared between PLT in stored PC before transfusion with the PLT-rich plasma (PRP) from the recipients after transfusion and with PRP from patients with moderate decrease in PLT counts after chemotherapy. RESULTS: The maximum extent of PLT aggregation was significantly higher in PRP collected from the patients after transfusion compared to the extent obtained before transfusion. There were no significant differences in the maximum extents of PLT aggregation between the PRP collected from the recipients after PC transfusion and, in the same patients, when PLT counts were moderately low. CONCLUSION: These results suggest that the observed decreased in PLT aggregation after storage can improve in the body after transfusion, and transfused PLTs have similar aggregation ability compared to the PLTs derived from the patient.