PMID- 35936184
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20220809
IS  - 2168-8184 (Print)
IS  - 2168-8184 (Electronic)
IS  - 2168-8184 (Linking)
VI  - 14
IP  - 7
DP  - 2022 Jul
TI  - Assessing the Efficacy of Alkylating Agent Regimens in the Treatment of Infantile 
      Malignant Osteopetrosis: Cyclophosphamide, Busulfan, or Thiotepa.
PG  - e26600
LID - 10.7759/cureus.26600 [doi]
LID - e26600
AB  - Infantile malignant osteopetrosis is a debilitating disease that requires total 
      bone marrow irradiation and transplant procedures for patients to survive. The 
      major complication of this procedure is graft vs host disease (GVHD), followed by 
      infections and end organ toxicity. Therefore, current research efforts into 
      treatment mainly aim to reduce GVHD while limiting infections and organ toxicity. 
      Different regimens of alkylating agents have been used to try to reduce GVHD. The 
      most common regimen is cyclophosphamide (Cy) with busulfan (Bu), followed by Cy 
      with Bu and thiotepa (Thio). This meta-analysis aimed to evaluate the efficacy of 
      different treatments by comparing mortality and morbidity causes and rates across 
      groups. The mean one-year survival rate for the Cy, Bu, Thio regimen studies in 
      the human leukocyte antigen (HLA) unmatched group (45.01%) was statistically 
      lower than the one-year survival rate for the studies using just a Cy, Bu regimen 
      (70.8%) in the HLA unmatched studies (p<0.00142). The one-year survival in the 
      studies which had HLA-matched donors was 80.56%, which is statistically higher 
      (p<0.001) than the one-year survival in the HLA-unmatched studies (53.96%), 
      indicating a benefit of finding HLA-matched donors. It seems that price and 
      availability could be a factor in the widespread use of Cy.
CI  - Copyright (c) 2022, Wagh et al.
FAU - Wagh, Himanshu
AU  - Wagh H
AD  - Medicine, California Northstate University College of Medicine, Elk Grove, USA.
FAU - Arif, Amber
AU  - Arif A
AD  - Medicine, California Northstate University College of Medicine, Elk Grove, USA.
FAU - Reddy, Akshay J
AU  - Reddy AJ
AD  - Ophthalmology, California University of Science and Medicine, Colton, USA.
FAU - Tabaie, Ethan
AU  - Tabaie E
AD  - Neurobiology, Physiology, and Behavior, University of California (UC), Davis, 
      USA.
FAU - Shekhar, Aditya
AU  - Shekhar A
AD  - Medicine, California Northstate University College of Medicine, Elk Grove, USA.
FAU - Min, Mildred
AU  - Min M
AD  - Medicine, California Northstate University College of Medicine, Elk Grove, USA.
FAU - Nawathey, Neel
AU  - Nawathey N
AD  - Health Sciences, California Northstate University, Rancho Cordova, USA.
FAU - Bachir, Mark
AU  - Bachir M
AD  - Health Sciences, California Northstate University, Rancho Cordova, USA.
FAU - Brahmbhatt, Hetal
AU  - Brahmbhatt H
AD  - Psychiatry, Mercy General Hospital, Sacramento, USA.
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20220706
PL  - United States
TA  - Cureus
JT  - Cureus
JID - 101596737
PMC - PMC9354912
OTO - NOTNLM
OT  - alkylating agents
OT  - busulfan
OT  - cyclophosphamide
OT  - graft vs host disease
OT  - hematopoiesis
OT  - infantile
OT  - meta-analysis
OT  - osteopetrosis
OT  - thiotepa
OT  - treatment choices
COIS- The authors have declared that no competing interests exist.
EDAT- 2022/08/09 06:00
MHDA- 2022/08/09 06:01
PMCR- 2022/07/06
CRDT- 2022/08/08 03:44
PHST- 2022/07/06 00:00 [accepted]
PHST- 2022/08/08 03:44 [entrez]
PHST- 2022/08/09 06:00 [pubmed]
PHST- 2022/08/09 06:01 [medline]
PHST- 2022/07/06 00:00 [pmc-release]
AID - 10.7759/cureus.26600 [doi]
PST - epublish
SO  - Cureus. 2022 Jul 6;14(7):e26600. doi: 10.7759/cureus.26600. eCollection 2022 Jul.