Fatal Cyclophosphamide-Induced Congestive Heart Failure in a 10-Year-Old Boy with Shwachman-Diamond Syndrome and Severe Bone Marrow Failure Treated with Allogeneic Bone Marrow Transplantation

Tsai, Philip H.; Sahdev, Indira; Herry, Angelica; Lipton, Jeffrey M.

doi:10.1097/00043426-199122000-00021

Cited by 28 publications

(48 citation statements)

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“…15,23,28,31 Savilahti and Rapola 31 reported significant cardiac dysfunction in patients with SDS even without exposure to CY. In their series of 16 Finnish patients, 8 had cardiac abnormalities on necropsy including cardiac fibrosis and areas of necrosis.…”

Section: Discussionmentioning

confidence: 99%

“…Additionally, patients with SDS tend to have increased toxicity with intensive conditioning regimens. Tsai et al 15 reported a case of fatal congestive heart failure following a Cy-containing-conditioning regimen. Other authors have described neurological complications, 16 pulmonary complications and multiorgan failure with typical ablative regimens.…”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Reduced-intensity conditioning is effective and safe for transplantation of patients with Shwachman–Diamond syndrome

Bhatla

Davies

Shenoy

et al. 2008

Bone Marrow Transplant

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Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Reduced-intensity conditioning is effective and safe for transplantation of patients with Shwachman–Diamond syndrome

Bhatla

Davies

Shenoy

et al. 2008

Bone Marrow Transplant

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“…Bone Marrow Transplantation opposed to Fanconi anemia, 31 Shwachman Diamond syndrome 32 and dyskeratosis congenita, 33 DBA in itself does not appear to pose a particular risk for allogeneic matched sibling SCT.…”

Section: Discussionmentioning

confidence: 99%

Hematopoietic stem cell transplantation for Diamond Blackfan anemia: a report from the Diamond Blackfan Anemia Registry

Vlachos

Federman

Reyes-Haley

et al. 2001

Bone Marrow Transplant

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Summary:The Diamond Blackfan Anemia (DBA) Registry of North America is a detailed database of patients with DBA from the United States and Canada. To date, 354 patients have been registered. From this database an analysis of the outcome of hematopoietic stem cell transplantation for DBA was undertaken. Of the 20 transplanted patients who met criteria for the diagnosis of DBA, eight underwent an allogeneic HLA-matched sibling hematopoietic stem cell transplant (SCT) and 12 an alternative donor SCT. The median age at transplant for all patients was 6 years 2 months; 3 years 10 months vs 9 years 1 month for HLA-matched sibling and alternative donor SCT, respectively. All of the HLA-matched sibling transplants were done using a non-irradiationcontaining regimen, whereas the majority of alternative donor transplants were performed using total body irradiation. The survival for HLA-matched sibling vs alternative donor transplant was 87.5% ؎ 11.7% vs 14.1% ؎ 12.1% at greater than 5 years from SCT (P ‫؍‬ 0.015). The use of HLA-matched sibling SCT should be considered for all patients with suitable donors. However, alternative donor SCT in DBA must be approached cautiously, the potential for severe aplastic anemia (SAA) or hematopoietic malignancy not withstanding. Bone Marrow Transplantation (2001) 27, 381-386.

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“…Including this report, eight cases of allogeneic BMT have been reported on SDS patients (see Table 1). [3][4][5][6][7][8] Severe to fatal complications were encountered in six patients posttransplant. A patient with aplastic anemia died of cyclophosphamide-associated cardiomyopathy.…”

Section: Discussionmentioning

confidence: 99%

“…A patient with aplastic anemia died of cyclophosphamide-associated cardiomyopathy. 3 Myocardial fibrosis and cardiomegaly have been described in SDS patients. 9 Two patients had poor engraftment and a third had delayed marrow recovery from prior antileukemic chemotherapy.…”

Section: Discussionmentioning

confidence: 99%

Bone marrow transplantation in Shwachman–Diamond syndrome: report of two cases and review of the literature

Okcu

Roberts

Chan

1998

Bone Marrow Transplant

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Summary:Patients with Shwachman-Diamond syndrome (SDS) have an increased frequency of myelodysplasia and leukemic transformation. We described two patients who received allogeneic stem cell transplantation and developed multiple complications, including seizure, hyperglycemia and renal tubular acidosis. A review of the literature showed that patients with SDS appeared to have an increased incidence of various transplantassociated problems. These patients frequently have underlying organ dysfunction and should be managed with extreme caution when treated with allogeneic stem cell transplantation. Keywords: Shwachman-Diamond syndrome; myelodysplasia; leukemia; BMT; complications Shwachman-Diamond syndrome (SDS) is a rare autosomal recessive disorder characterized by exocrine insufficiency, short stature, skeletal abnormalities and bone marrow dysfunction. There is an increased risk of marrow aplasia and leukemic transformation, the latter often preceded by a phase of myelodysplasia (MDS). 1,2 Allogeneic stem cell transplantation offers potentially curative treatment for MDS, leukemia and marrow failure syndromes. Few patients with SDS have undergone BMT. We report our experience of allogeneic BMT in two SDS patients who developed MDS and monosomy 7 abnormality. Severe complications were encountered in both cases. We reviewed the outcome of allotransplantation in other patients published in the literature and noticed a significant number of post-transplant problems. Case 1An 8-year-old boy with MDS/monosomy 7 had a past history of short stature, growth hormone deficiency, recurrent diarrhea and loss of mineralization of distal femora. CT scan of the abdomen showed a fatty pancreas and serum amylase and lipase activity were virtually absent. He received a CD5, CD8 T cell-depleted (Applied Immune Sciences, Santa Clara, CA, USA), unrelated donor (micromismatched at 1 HLA-DR locus) BMT after conditioning with thiotepa 5 mg/kg i.v. on day −7, CY 60 mg/kg i.v. on day −6, −5 and TBI 3 Gy/day on days −4 to −1. GVHD prophylaxis included CsA, short Mtx and MP. The post-BMT course was complicated by several episodes of seizure of unknown etiology. Later on he developed severe electrolyte wasting as the result of renal tubular acidosis. He had grade IV GVHD that failed to respond to MP, antithymocyte globulin and hydroxychloroquine therapy. He became severely hyperglycemic (blood sugar Ͼ1000 mg%) and hypernatremic after pulse MP therapy. This responded slowly to insulin and hydration. He died on day 93 of pulmonary hemorrhage. An autopsy was not performed. His ANC exceeded 500/mm 3 on day 18 but he remained platelet and red cell transfusion-dependent. A bone marrow examination on day 31 showed 1.2% blasts and normal cytogenetics. Case 2A 9-year-old child with a history of short stature, growth hormone deficiency, and metaphyseal chondroplasia developed marrow hypoplasia. Bone marrow examination 2 years later revealed myelofibrosis, with monosomy 7 and + i(7q) cytogenetic abnormalities. A MRI of the abdomen showed hemosiderosis...

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Fatal Cyclophosphamide-Induced Congestive Heart Failure in a 10-Year-Old Boy with Shwachman-Diamond Syndrome and Severe Bone Marrow Failure Treated with Allogeneic Bone Marrow Transplantation

Cited by 28 publications

References 0 publications

Reduced-intensity conditioning is effective and safe for transplantation of patients with Shwachman–Diamond syndrome

Reduced-intensity conditioning is effective and safe for transplantation of patients with Shwachman–Diamond syndrome

Hematopoietic stem cell transplantation for Diamond Blackfan anemia: a report from the Diamond Blackfan Anemia Registry

Bone marrow transplantation in Shwachman–Diamond syndrome: report of two cases and review of the literature

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