Allogeneic hematopoietic cell transplantation using fludarabine plus myeloablative busulfan and melphalan confers promising survival in high-risk hematopoietic neoplasms: a single-center retrospective analysis

Edahiro, Taro; Kawase, Takakazu; Nagoshi, Hisao; Fujino, Keita; Toishigawa, Kayo; Miyama, T.; Mino, Tatsuji; Yoshida, Toyonobu; Morioka, Takehiko; Hirata, Yuji; Noma, Mitsunori; Fujii, Teruhisa; Nishizawa, Masatoshi; Fukushima, Noriyasu; Ichinohe, Tatsuo

doi:10.1080/16078454.2021.1881228

Cited by 5 publications

(5 citation statements)

References 40 publications

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“…The authors concluded that this was a safe and successful regimen considering the highest risk group included many chemo-resistant patients with active disease at the time of transplant. 30 We observed a significantly higher incidence of grade II-IV aGvHD in MSD and MUD PBSCT compared to patients undergoing haplo-BMT (Figure 1B). As expected with PBSC grafts, we saw a significantly increased cumulative incidence of cGvHD (one mild, two moderate, and one severe) and MUD allo-HCT (one moderate and one severe)…”

Section: Discussionmentioning

confidence: 67%

“…Similarly, a retrospective single‐center review of 39 adult patients reported on their use of FLU‐BU‐MEL in myeloid and lymphoid malignancies with BM, PB, and UCB as stem cell sources, with a 2‐year OS of 62% for their entire cohort and 44% in their high‐risk to very high‐risk subset. The authors concluded that this was a safe and successful regimen considering the highest risk group included many chemo‐resistant patients with active disease at the time of transplant 30 …”

Section: Discussionmentioning

confidence: 99%

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Busulfan, fludarabine, and melphalan are effective conditioning for pediatric and young adult patients with myeloid malignancies underdoing matched sibling or alternative donor transplantation

Truscott

Pariury

Hanmod

et al. 2022

Pediatric Blood & Cancer

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Background Allogeneic hematopoietic cell transplantation (allo‐HCT) remains a curative option for patients with high‐risk myeloid malignancies. Procedure We present our 10‐year experience (October 2012 to October 2021) of consecutive allo‐HCT in patients with myeloid malignancies treated on the pediatric HCT service and conditioned with myeloablative targeted dose—busulfan (BU), fludarabine (FLU), and melphalan (MEL). Twenty‐three children, adolescents, and young adult patients (CAYA) (median age 15.4 years) with acute myeloid leukemia (AML, n = 17), myelodysplastic syndrome (MDS, n = 4), or chronic myeloid leukemia (CML, n = 2) underwent allo‐HCT post‐BU‐FLU‐MEL. Four patients had treatment‐related AML/MDS. Donor/stem cell source was matched sibling donor (MSD) PBSC (n = 7), matched unrelated donor (MUD) PBSC (n = 2), umbilical cord blood (UCB) (n = 3), or haploidentical‐BMT (n = 11). Risk stratification was low (n = 2), intermediate (n = 15), high (n = 3), and very high risk (n = 1). The two patients with CML had failed tyrosine kinase inhibitor therapies. Results With a median follow‐up of 41.6 months, the relapse rate is only 4.5% with an overall survival (OS) 100%, progression‐free survival (PFS) 95.5%, and graft‐versus‐host‐free‐relapse‐free survival (GRFS) 67.8%. The donor source and the acute graft‐versus‐host disease (GvHD) prophylaxis regimen significantly impacted grade II–IV aGvHD 66.7% versus 19.2% (p = .039) and chronic graft‐versus‐host‐disease (cGvHD) 66.7% versus 0% (p = .002) in the patients receiving MSD or MUD PBSC compared to haplo‐BMT, respectively, resulting in improved GRFS in haplo‐BMT, 83.3% compared to 40% matched donor peripheral blood stem cell transplant (PBSCT) (p = .025). Conclusions Our results demonstrate that BU‐FLU‐MEL is efficacious conditioning for disease control in young patients with myeloid malignancies undergoing MSD or alternative donor allo‐HCT, but in the setting of PBSC grafts with cyclosporine A‐methotrexate (CSA‐MTX) GvHD prophylaxis, it results in an unacceptably high incidence of GvHD.

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Section: Discussionmentioning

confidence: 67%

Section: Discussionmentioning

confidence: 99%

Busulfan, fludarabine, and melphalan are effective conditioning for pediatric and young adult patients with myeloid malignancies underdoing matched sibling or alternative donor transplantation

Truscott

Pariury

Hanmod

et al. 2022

Pediatric Blood & Cancer

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“…On the other hand, 12 patients (57.1%) were treated under the Myeloablative Conditioning (MAC) regimen, which employed more intensive regimens than RIC. Specifically, in the MAC regimen, combinations such as fludarabine 30 mg/m 2 /d × 4 days plus busulfan 3.2 mg/kg/d × 4 days (FluBu4) or FluBu4 and Mel 140 mg/m 2 × 1 day (FluBu4-Mel140) were utilized [21]. In autologous transplantation, other MAC regimens like melphalan 100 mg/m 2 /d × 2 days (Mel200) and the MEAM protocol, consisting of the combination of ranimustine (MCNU) 300 mg/m 2 /d, etoposide 200 mg/m 2 /d × 4 days, cytarabine 400 mg/m 2 /d, and melphalan 140 mg/m 2 /d, were also used.…”

Section: Patient Characteristicsmentioning

confidence: 99%

Efficacy of Low-Level Laser Therapy for Oral Mucositis in Hematologic Patients Undergoing Transplantation: A Single-Arm Prospective Study

Nishi,

Horikoshi,

Ohta

et al. 2023

JPM

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Oral mucositis significantly affects the quality of life in hematologic cancer patients undergoing hematopoietic stem cell transplantation. Despite global evidence supporting the efficacy of low-level laser therapy (LLLT) for mucositis prevention, its clinical adoption in Japan is limited. This study aimed to fill this gap by evaluating the safety and efficacy of LLLT in a Japanese patient population. In a single-group, non-blinded, exploratory trial, we compared 21 LLLT-treated patients against a historical control of 96 patients. The primary endpoint was the incidence of Grade ≥ 2 mucositis, based on NCI-CTCAE ver. 4.0. The LLLT group showed a significantly lower incidence of Grade ≥ 2 mucositis (23.8%) compared to the control group (64.6%) (p = 0.0006). Furthermore, Grade ≥ 2 mucositis correlated with increased oral dryness and longer hospital stays. Our study confirms the efficacy of LLLT in reducing the onset of severe oral mucositis among Japanese hematologic cancer patients, advocating for its clinical introduction as a preventive measure in Japan.

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“…Giteritinib was continued until three days before conditioning ( Fig. 1 ) with a myeloablative conditioning regimen of fludarabine (30 mg/m 2 ; from day -6 to day -3), busulfan (3.2 mg/kg; from day -6 to day -3), and melphalan (140 mg/m 2 ; day -2) ( 18 ) followed by infusion of peripheral blood stem cells containing 1.12×10 6 /kg CD34-positive cells. Graft-versus-host disease (GVHD) prophylaxis included cyclosporine (from day -1) and methotrexate (10 mg/m 2 on day +1 followed by 7 mg/m 2 on day +3, +6 and +11).…”

Section: Case Reportmentioning

confidence: 99%

Successful Pre- and Post-transplant Administration of Gilteritinib in a Patient with Relapsed and Refractory Acute Myeloid Leukemia Undergoing Allogeneic Peripheral Blood Stem Cell Transplantation

et al. 2023

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Patients with acute myeloid leukemia (AML) harboring FMS-like tyrosine kinase 3 (FLT3)-internal tandem duplication mutation are associated with a poor survival outcome, even those receiving allogeneic stem cell transplantation (Allo-SCT). An additional treatment strategy with allo-SCT is therefore required to reduce relapse in these patients. Gilteritinib is a specific FLT3 inhibitor that has shown clinical benefit for patients with relapsed and refractory (R/R) AML harboring FLT3 mutation. We herein report a 49-year-old woman with R/R AML who was successfully treated with pre-and post-transplant gilteritinib. Post-transplant gilteritnib yielded a durable response with possible exacerbation of graft-versus-host disease.

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Allogeneic hematopoietic cell transplantation using fludarabine plus myeloablative busulfan and melphalan confers promising survival in high-risk hematopoietic neoplasms: a single-center retrospective analysis

Cited by 5 publications

References 40 publications

Busulfan, fludarabine, and melphalan are effective conditioning for pediatric and young adult patients with myeloid malignancies underdoing matched sibling or alternative donor transplantation

Busulfan, fludarabine, and melphalan are effective conditioning for pediatric and young adult patients with myeloid malignancies underdoing matched sibling or alternative donor transplantation

Efficacy of Low-Level Laser Therapy for Oral Mucositis in Hematologic Patients Undergoing Transplantation: A Single-Arm Prospective Study

Successful Pre- and Post-transplant Administration of Gilteritinib in a Patient with Relapsed and Refractory Acute Myeloid Leukemia Undergoing Allogeneic Peripheral Blood Stem Cell Transplantation

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